DE69123979T2 - Abgeänderte ribozyme - Google Patents

Abgeänderte ribozyme

Info

Publication number
DE69123979T2
DE69123979T2 DE69123979T DE69123979T DE69123979T2 DE 69123979 T2 DE69123979 T2 DE 69123979T2 DE 69123979 T DE69123979 T DE 69123979T DE 69123979 T DE69123979 T DE 69123979T DE 69123979 T2 DE69123979 T2 DE 69123979T2
Authority
DE
Germany
Prior art keywords
modified
ribozyms
amino
rna molecules
modified rna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Expired - Fee Related
Application number
DE69123979T
Other languages
English (en)
Other versions
DE69123979D1 (de
Inventor
Fritz Eckstein
Wolfgang Pieken
Fritz Benseler
David Olsen
David Williams
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Max Planck Gesellschaft zur Foerderung der Wissenschaften eV
Original Assignee
Max Planck Gesellschaft zur Foerderung der Wissenschaften eV
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Max Planck Gesellschaft zur Foerderung der Wissenschaften eV filed Critical Max Planck Gesellschaft zur Foerderung der Wissenschaften eV
Publication of DE69123979D1 publication Critical patent/DE69123979D1/de
Application granted granted Critical
Publication of DE69123979T2 publication Critical patent/DE69123979T2/de
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1131Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses
    • C12N15/1132Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against viruses against retroviridae, e.g. HIV
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • A61P31/14Antivirals for RNA viruses
    • A61P31/18Antivirals for RNA viruses for HIV
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/121Hammerhead
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/12Type of nucleic acid catalytic nucleic acids, e.g. ribozymes
    • C12N2310/122Hairpin
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
DE69123979T 1990-10-12 1991-09-23 Abgeänderte ribozyme Expired - Fee Related DE69123979T2 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP9001731 1990-10-12
PCT/EP1991/001811 WO1992007065A1 (en) 1990-10-12 1991-09-23 Modified ribozymes

Publications (2)

Publication Number Publication Date
DE69123979D1 DE69123979D1 (de) 1997-02-13
DE69123979T2 true DE69123979T2 (de) 1997-04-30

Family

ID=8165527

Family Applications (2)

Application Number Title Priority Date Filing Date
DE91916858T Pending DE552178T1 (de) 1990-10-12 1991-09-23 Abgeänderte ribozyme.
DE69123979T Expired - Fee Related DE69123979T2 (de) 1990-10-12 1991-09-23 Abgeänderte ribozyme

Family Applications Before (1)

Application Number Title Priority Date Filing Date
DE91916858T Pending DE552178T1 (de) 1990-10-12 1991-09-23 Abgeänderte ribozyme.

Country Status (11)

Country Link
US (3) US5672695A (de)
EP (1) EP0552178B1 (de)
JP (2) JP3257675B2 (de)
AT (1) ATE147098T1 (de)
AU (1) AU649074B2 (de)
CA (1) CA2093664C (de)
DE (2) DE552178T1 (de)
ES (1) ES2061416T3 (de)
GR (1) GR930300130T1 (de)
PL (1) PL169576B1 (de)
WO (1) WO1992007065A1 (de)

Families Citing this family (462)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5948634A (en) * 1988-12-21 1999-09-07 The General Hospital Coporation Neural thread protein gene expression and detection of alzheimer's disease
US5872232A (en) * 1990-01-11 1999-02-16 Isis Pharmaceuticals Inc. 2'-O-modified oligonucleotides
US5859221A (en) * 1990-01-11 1999-01-12 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US6005087A (en) * 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-modified oligonucleotides
US5670633A (en) * 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5955589A (en) * 1991-12-24 1999-09-21 Isis Pharmaceuticals Inc. Gapped 2' modified oligonucleotides
US5623065A (en) * 1990-08-13 1997-04-22 Isis Pharmaceuticals, Inc. Gapped 2' modified oligonucleotides
US6399754B1 (en) 1991-12-24 2002-06-04 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides
US6395888B1 (en) * 1996-02-01 2002-05-28 Gilead Sciences, Inc. High affinity nucleic acid ligands of complement system proteins
AU650032B2 (en) * 1990-06-19 1994-06-09 Gene Shears Pty. Limited Endonucleases
AU649074B2 (en) * 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
US7015315B1 (en) 1991-12-24 2006-03-21 Isis Pharmaceuticals, Inc. Gapped oligonucleotides
US5965722A (en) * 1991-05-21 1999-10-12 Isis Pharmaceuticals, Inc. Antisense inhibition of ras gene with chimeric and alternating oligonucleotides
US6307040B1 (en) 1992-03-05 2001-10-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US7119184B2 (en) * 1991-08-12 2006-10-10 Isis Pharmaceuticals, Inc. Oligonucleotides having A-DNA form and B-DNA form conformational geometry
DK1695979T3 (da) * 1991-12-24 2011-10-10 Isis Pharmaceuticals Inc Gappede modificerede oligonukleotider
US6277603B1 (en) 1991-12-24 2001-08-21 Isis Pharmaceuticals, Inc. PNA-DNA-PNA chimeric macromolecules
US5856455A (en) * 1991-12-24 1999-01-05 Isis Pharmaceuticals, Inc. Gapped 2'-modified oligonucleotides
US5652094A (en) * 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
US6204027B1 (en) * 1992-02-26 2001-03-20 University Of Massachusetts Worcester Ribozymes having 2′-O substituted nucleotides in the flanking sequences
US6469158B1 (en) 1992-05-14 2002-10-22 Ribozyme Pharmaceuticals, Incorporated Synthesis, deprotection, analysis and purification of RNA and ribozymes
WO2002081494A1 (en) * 2001-03-26 2002-10-17 Sirna Therapeutics, Inc. Oligonucleotide mediated inhibition of hepatitis b virus and hepatitis c virus replication
US20030206887A1 (en) * 1992-05-14 2003-11-06 David Morrissey RNA interference mediated inhibition of hepatitis B virus (HBV) using short interfering nucleic acid (siNA)
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
US5804683A (en) * 1992-05-14 1998-09-08 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA with alkylamine
US20040127446A1 (en) * 1992-05-14 2004-07-01 Lawrence Blatt Oligonucleotide mediated inhibition of hepatitis B virus and hepatitis C virus replication
US20040054156A1 (en) * 1992-05-14 2004-03-18 Kenneth Draper Method and reagent for inhibiting hepatitis B viral replication
US5686599A (en) * 1992-05-14 1997-11-11 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
US20030125270A1 (en) * 2000-12-18 2003-07-03 Lawrence Blatt Enzymatic nucleic acid treatment of diseases or conditions related to hepatitis C virus infection
US6346614B1 (en) 1992-07-23 2002-02-12 Hybridon, Inc. Hybrid oligonucleotide phosphorothioates
TW244371B (de) * 1992-07-23 1995-04-01 Tri Clover Inc
US5652355A (en) * 1992-07-23 1997-07-29 Worcester Foundation For Experimental Biology Hybrid oligonucleotide phosphorothioates
US5646042A (en) * 1992-08-26 1997-07-08 Ribozyme Pharmaceuticals, Inc. C-myb targeted ribozymes
US5891684A (en) * 1992-10-15 1999-04-06 Ribozyme Pharmaceuticals, Inc. Base-modified enzymatic nucleic acid
US5864028A (en) * 1992-11-03 1999-01-26 Gene Shears Pty. Limited Degradation resistant mRNA derivatives linked to TNF-α ribozymes
JPH08505521A (ja) * 1992-11-03 1996-06-18 ジーン・シアーズ・プロプライアタリー・リミテッド TNF−αリボザイムおよびTNF−αリボザイムにリンクした耐分解性mRNA誘導体
GB9225427D0 (en) * 1992-12-04 1993-01-27 Ribonetics Gmbh Oligonucleotides with rna cleavage activity
US5612215A (en) * 1992-12-07 1997-03-18 Ribozyme Pharmaceuticals, Inc. Stromelysin targeted ribozymes
US5811300A (en) * 1992-12-07 1998-09-22 Ribozyme Pharmaceuticals, Inc. TNF-α ribozymes
US5616488A (en) * 1992-12-07 1997-04-01 Ribozyme Pharmaceuticals, Inc. IL-5 targeted ribozymes
US5837542A (en) * 1992-12-07 1998-11-17 Ribozyme Pharmaceuticals, Inc. Intercellular adhesion molecule-1 (ICAM-1) ribozymes
US5658780A (en) * 1992-12-07 1997-08-19 Ribozyme Pharmaceuticals, Inc. Rel a targeted ribozymes
US5817635A (en) * 1993-08-09 1998-10-06 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
EP0748382B1 (de) 1993-09-02 2002-11-06 Ribozyme Pharmaceuticals, Inc. Enzymatische nukleiksaüre die nicht-nukleotide enthaltet
WO1995006764A2 (en) * 1993-09-03 1995-03-09 Vpi Holdings Ltd. Oligonucleotides with rna cleavage activity
US5861288A (en) * 1993-10-18 1999-01-19 Ribozyme Pharmaceuticals, Inc. Catalytic DNA
AU8088694A (en) * 1993-10-27 1995-05-22 Ribozyme Pharmaceuticals, Inc. 2'-amido and 2'-peptido modified oligonucleotides
AU730347B2 (en) * 1993-11-08 2001-03-08 Ribozyme Pharmaceuticals, Inc. Base-modified enzymatic nucleic acid
AU687479B2 (en) * 1993-11-08 1998-02-26 Ribozyme Pharmaceuticals, Inc. Base-modified enzymatic nucleic acid
US6897016B1 (en) 1993-11-12 2005-05-24 The Regents Of The University Of Colorado Alteration of sequence of a target molecule
US5631359A (en) * 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
AU706417B2 (en) * 1994-02-23 1998-06-17 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting the expression of disease related genes
US5639647A (en) * 1994-03-29 1997-06-17 Ribozyme Pharmaceuticals, Inc. 2'-deoxy-2'alkylnucleotide containing nucleic acid
US5693532A (en) * 1994-11-04 1997-12-02 Ribozyme Pharmaceuticals, Inc. Respiratory syncytial virus ribozymes
US5902880A (en) * 1994-08-19 1999-05-11 Ribozyme Pharmaceuticals, Inc. RNA polymerase III-based expression of therapeutic RNAs
EP1502950A3 (de) * 1994-02-23 2005-06-22 Ribozyme Pharmaceuticals, Inc. Verfahren zur Reinigung von chemisch-modifiziertem RNA
US5627053A (en) * 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US6103890A (en) * 1994-05-18 2000-08-15 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids that cleave C-fos
US5633133A (en) * 1994-07-14 1997-05-27 Long; David M. Ligation with hammerhead ribozymes
US6146886A (en) 1994-08-19 2000-11-14 Ribozyme Pharmaceuticals, Inc. RNA polymerase III-based expression of therapeutic RNAs
US5599706A (en) * 1994-09-23 1997-02-04 Stinchcomb; Dan T. Ribozymes targeted to apo(a) mRNA
US5700923A (en) * 1994-09-29 1997-12-23 Hybridon, Inc. Finderons and methods of their preparation and use
US5716824A (en) * 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
US5672501A (en) * 1994-12-23 1997-09-30 Ribozyme Pharmaceuticals, Inc. Base-modified enzymatic nucleic acid
US5545729A (en) * 1994-12-22 1996-08-13 Hybridon, Inc. Stabilized ribozyme analogs
US5705388A (en) * 1994-12-23 1998-01-06 Ribozyme Pharmaceuticals, Inc. CETP Ribozymes
US6017887A (en) * 1995-01-06 2000-01-25 Sibia Neurosciences, Inc. Peptide, peptide analog and amino acid analog protease inhibitors
US5804560A (en) * 1995-01-06 1998-09-08 Sibia Neurosciences, Inc. Peptide and peptide analog protease inhibitors
US5663064A (en) * 1995-01-13 1997-09-02 University Of Vermont Ribozymes with RNA protein binding site
US5877021A (en) 1995-07-07 1999-03-02 Ribozyme Pharmaceuticals, Inc. B7-1 targeted ribozymes
US7034009B2 (en) * 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US20040142895A1 (en) * 1995-10-26 2004-07-22 Sirna Therapeutics, Inc. Nucleic acid-based modulation of gene expression in the vascular endothelial growth factor pathway
US20040102389A1 (en) * 1995-10-26 2004-05-27 Ribozyme Pharmaceuticals, Inc. Nucleic acid-mediated treatment of diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US20040220128A1 (en) * 1995-10-26 2004-11-04 Sirna Therapeutics, Inc. Nucleic acid based modulation of female reproductive diseases and conditions
US6346398B1 (en) 1995-10-26 2002-02-12 Ribozyme Pharmaceuticals, Inc. Method and reagent for the treatment of diseases or conditions related to levels of vascular endothelial growth factor receptor
AU7572096A (en) * 1995-11-14 1997-06-05 Vimrx Holdings, Ltd. Chimeric oligomers having an rna-cleavage activity
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
PT808898E (pt) * 1996-05-24 2004-10-29 Aventis Pharma Gmbh Reagente e metodo para inibicao da expressao de n-ras
US20030044941A1 (en) 1996-06-06 2003-03-06 Crooke Stanley T. Human RNase III and compositions and uses thereof
US7812149B2 (en) 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US5898031A (en) * 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US5807743A (en) * 1996-12-03 1998-09-15 Ribozyme Pharmaceuticals, Inc. Interleukin-2 receptor gamma-chain ribozymes
ATE398139T1 (de) * 1996-12-13 2008-07-15 Novartis Vaccines & Diagnostic Analyse und separation von pdgf-proteinen
EP0958303A4 (de) * 1996-12-19 2004-03-31 Univ Yale Bioreaktive allosterische polynukleotide
US6248878B1 (en) 1996-12-24 2001-06-19 Ribozyme Pharmaceuticals, Inc. Nucleoside analogs
US6057156A (en) 1997-01-31 2000-05-02 Robozyme Pharmaceuticals, Inc. Enzymatic nucleic acid treatment of diseases or conditions related to levels of epidermal growth factor receptors
US20030186909A1 (en) * 1997-01-27 2003-10-02 Ribozyme Pharmaceuticals, Inc. Nucleic acid treatment of diseases or conditions related to levels of epidermal growth factor receptors
US6159951A (en) * 1997-02-13 2000-12-12 Ribozyme Pharmaceuticals Inc. 2'-O-amino-containing nucleoside analogs and polynucleotides
WO1998038204A1 (en) 1997-02-26 1998-09-03 The General Hospital Corporation Transgenic animals and cell lines for screening drugs effective for the treatment or prevention of alzheimer's disease
US7226730B1 (en) 1997-02-26 2007-06-05 The General Hospital Corporation Transgenic animals and cell lines for screening drugs effective for the treatment or prevention of Alzheimer's Disease
US6251666B1 (en) 1997-03-31 2001-06-26 Ribozyme Pharmaceuticals, Inc. Nucleic acid catalysts comprising L-nucleotide analogs
US6280936B1 (en) 1997-06-09 2001-08-28 Ribozyme Pharmaceuticals, Inc. Method for screening nucleic acid catalysts
US6548657B1 (en) 1997-06-09 2003-04-15 Ribozyme Pharmaceuticals, Inc. Method for screening nucleic acid catalysts
US6183959B1 (en) 1997-07-03 2001-02-06 Ribozyme Pharmaceuticals, Inc. Method for target site selection and discovery
US6316612B1 (en) 1997-08-22 2001-11-13 Ribozyme Pharmaceuticals, Inc. Xylofuranosly-containing nucleoside phosphoramidites and polynucleotides
US6656731B1 (en) 1997-09-22 2003-12-02 Max Planck Gesellschaft Zur Forderung Der Wissenschaften E.V. Nucleic acid catalysts with endonuclease activity
AU750947C (en) * 1997-09-22 2003-05-22 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Nucleic acid catalysts with endonuclease activity
US6054576A (en) 1997-10-02 2000-04-25 Ribozyme Pharmaceuticals, Inc. Deprotection of RNA
US6617438B1 (en) 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
US6127535A (en) * 1997-11-05 2000-10-03 Ribozyme Pharmaceuticals, Inc. Nucleoside triphosphates and their incorporation into oligonucleotides
US6482932B1 (en) * 1997-11-05 2002-11-19 Ribozyme Pharmaceuticals, Incorporated Nucleoside triphosphates and their incorporation into oligonucleotides
US20020147143A1 (en) 1998-03-18 2002-10-10 Corixa Corporation Compositions and methods for the therapy and diagnosis of lung cancer
JP2003525017A (ja) 1998-04-20 2003-08-26 リボザイム・ファーマシューティカルズ・インコーポレーテッド 遺伝子発現を調節しうる新規な化学組成を有する核酸分子
CA2230203A1 (en) 1998-04-29 1999-10-29 Universite De Sherbrooke Delta ribozyme for rna cleavage
US20030125269A1 (en) * 1998-08-26 2003-07-03 Ming Li T-type calcium channel
CA2340586A1 (en) * 1998-08-26 2000-03-23 South Alabama Medical Science Foundation T-type calcium channel
US6175004B1 (en) * 1998-09-01 2001-01-16 Isis Pharmaceuticals, Inc. Process for the synthesis of oligonucleotides incorporating 2-aminoadenosine
IL126732A0 (en) * 1998-10-23 1999-08-17 Intelligene Ltd Pro drug
US6995259B1 (en) 1998-10-23 2006-02-07 Sirna Therapeutics, Inc. Method for the chemical synthesis of oligonucleotides
AU4803800A (en) 1999-04-27 2000-11-10 Trustees Of The University Of Pennsylvania, The Compositions, methods, and kits relating to LTiGTresistinLT/iGT
EP1183389A2 (de) 1999-04-30 2002-03-06 University of Florida Ribozym zusamensetzungen verabreicht durch adeno asozierte viren und dessen verwendungen
US6617442B1 (en) * 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
US6831171B2 (en) 2000-02-08 2004-12-14 Yale University Nucleic acid catalysts with endonuclease activity
US20080039414A1 (en) * 2002-02-20 2008-02-14 Sima Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
US8202979B2 (en) * 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US20070026394A1 (en) * 2000-02-11 2007-02-01 Lawrence Blatt Modulation of gene expression associated with inflammation proliferation and neurite outgrowth using nucleic acid based technologies
US20050032733A1 (en) * 2001-05-18 2005-02-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA)
US20050233329A1 (en) * 2002-02-20 2005-10-20 Sirna Therapeutics, Inc. Inhibition of gene expression using duplex forming oligonucleotides
US8273866B2 (en) 2002-02-20 2012-09-25 Merck Sharp & Dohme Corp. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
AU3811101A (en) * 2000-02-11 2001-08-20 Lawrence Blatt Method and reagent for the modulation and diagnosis of cd20 and nogo gene expression
US6846486B1 (en) 2000-02-24 2005-01-25 Advanced Biotherapy Concepts, Inc. Method of treating allergy by administering an anti-histamine antibody
US20040002068A1 (en) 2000-03-01 2004-01-01 Corixa Corporation Compositions and methods for the detection, diagnosis and therapy of hematological malignancies
US20040023292A1 (en) * 2001-10-19 2004-02-05 Mcswiggen James Method and reagent for the detection of proteins and peptides
US20030065155A1 (en) * 2000-03-06 2003-04-03 Nassim Usman Nucleic acid sensor molecules
US20040009510A1 (en) * 2000-03-06 2004-01-15 Scott Seiwert Allosteric nucleic acid sensor molecules
ATE450621T2 (de) 2000-03-30 2009-12-15 Whitehead Biomedical Inst Mediatoren von rns-interferenz, die rns- sequenzspezifisch sind
EP1961819A3 (de) 2000-06-28 2008-11-12 Corixa Corporation Zusammensetzungen und Verfahren zur Heilung und Diagnose von Lungenkrebs
US20080032942A1 (en) * 2000-08-30 2008-02-07 Mcswiggen James RNA interference mediated treatment of Alzheimer's disease using short interfering nucleic acid (siNA)
US20050209179A1 (en) * 2000-08-30 2005-09-22 Sirna Therapeutics, Inc. RNA interference mediated treatment of Alzheimer's disease using short interfering nucleic acid (siNA)
US20030190635A1 (en) * 2002-02-20 2003-10-09 Mcswiggen James A. RNA interference mediated treatment of Alzheimer's disease using short interfering RNA
US20040259247A1 (en) * 2000-12-01 2004-12-23 Thomas Tuschl Rna interference mediating small rna molecules
US7049059B2 (en) * 2000-12-01 2006-05-23 Response Genetics, Inc. Method of determining a chemotherapeutic regimen based on ERCC1 and TS expression
ES2328796T3 (es) 2001-03-14 2009-11-18 Myriad Genetics, Inc. Interaccion tsg101-gag y uso de la misma.
WO2002078643A2 (en) 2001-03-30 2002-10-10 Philadelphia Health And Education Corporation Immunomodulation and effect on cell processes relating to serotonin family receptors
EP1515982A4 (de) 2001-05-09 2005-10-26 Corixa Corp Zusammensetzungen und verfahren zur therapie und diagnose von prostatakrebs
US20050080031A1 (en) * 2001-05-18 2005-04-14 Sirna Therapeutics, Inc. Nucleic acid treatment of diseases or conditions related to levels of Ras, HER2 and HIV
US20050148530A1 (en) * 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20060241075A1 (en) * 2001-05-18 2006-10-26 Sirna Therapeutics, Inc. RNA interference mediated inhibition of desmoglein gene expression using short interfering nucleic acid (siNA)
US20050227935A1 (en) * 2001-05-18 2005-10-13 Sirna Therapeutics, Inc. RNA interference mediated inhibition of TNF and TNF receptor gene expression using short interfering nucleic acid (siNA)
US20050256068A1 (en) * 2001-05-18 2005-11-17 Sirna Therapeutics, Inc. RNA interference mediated inhibition of stearoyl-CoA desaturase (SCD) gene expression using short interfering nucleic acid (siNA)
US20050054598A1 (en) * 2002-02-20 2005-03-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition hairless (HR) gene expression using short interfering nucleic acid (siNA)
US20050164224A1 (en) * 2001-05-18 2005-07-28 Sirna Therapeutics, Inc. RNA interference mediated inhibition of cyclin D1 gene expression using short interfering nucleic acid (siNA)
US20070042983A1 (en) * 2001-05-18 2007-02-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US20060142226A1 (en) * 2001-05-18 2006-06-29 Sirna Therapeutics, Inc. RNA interference mediated inhibition of cholesteryl ester transfer protein (CETP) gene expression using short interfering nucleic acid (siNA)
US20050233996A1 (en) * 2002-02-20 2005-10-20 Sirna Therapeutics, Inc. RNA interference mediated inhibition of hairless (HR) gene expression using short interfering nucleic acid (siNA)
US20050267058A1 (en) * 2001-05-18 2005-12-01 Sirna Therapeutics, Inc. RNA interference mediated inhibition of placental growth factor gene expression using short interfering nucleic acid (sINA)
US20040209831A1 (en) * 2002-02-20 2004-10-21 Mcswiggen James RNA interference mediated inhibition of hepatitis C virus (HCV) gene expression using short interfering nucleic acid (siNA)
US20050164966A1 (en) * 2001-05-18 2005-07-28 Sirna Therapeutics, Inc. RNA interference mediated inhibition of type 1 insulin-like growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20060211642A1 (en) * 2001-05-18 2006-09-21 Sirna Therapeutics, Inc. RNA inteference mediated inhibition of hepatitis C virus (HVC) gene expression using short interfering nucleic acid (siNA)
US20050119211A1 (en) * 2001-05-18 2005-06-02 Sirna Therapeutics, Inc. RNA mediated inhibition connexin gene expression using short interfering nucleic acid (siNA)
US20050176666A1 (en) * 2001-05-18 2005-08-11 Sirna Therapeutics, Inc. RNA interference mediated inhibition of GPRA and AAA1 gene expression using short interfering nucleic acid (siNA)
US20050287128A1 (en) * 2001-05-18 2005-12-29 Sirna Therapeutics, Inc. RNA interference mediated inhibition of TGF-beta and TGF-beta receptor gene expression using short interfering nucleic acid (siNA)
US20050130181A1 (en) * 2001-05-18 2005-06-16 Sirna Therapeutics, Inc. RNA interference mediated inhibition of wingless gene expression using short interfering nucleic acid (siNA)
US20050124567A1 (en) * 2001-05-18 2005-06-09 Sirna Therapeutics, Inc. RNA interference mediated inhibition of TRPM7 gene expression using short interfering nucleic acid (siNA)
US20040006035A1 (en) * 2001-05-29 2004-01-08 Dennis Macejak Nucleic acid mediated disruption of HIV fusogenic peptide interactions
US20050159379A1 (en) * 2001-05-18 2005-07-21 Sirna Therapeutics, Inc RNA interference mediated inhibition of gastric inhibitory polypeptide (GIP) and gastric inhibitory polypeptide receptor (GIPR) gene expression using short interfering nucleic acid (siNA)
US20050079610A1 (en) * 2001-05-18 2005-04-14 Sirna Therapeutics, Inc. RNA interference mediated inhibition of Fos gene expression using short interfering nucleic acid (siNA)
US20050153914A1 (en) * 2001-05-18 2005-07-14 Sirna Therapeutics, Inc. RNA interference mediated inhibition of MDR P-glycoprotein gene expression using short interfering nucleic acid (siNA)
US20060142225A1 (en) * 2001-05-18 2006-06-29 Sirna Therapeutics, Inc. RNA interference mediated inhibition of cyclin dependent kinase-2 (CDK2) gene expression using short interfering nucleic acid (siNA)
US20050164968A1 (en) * 2001-05-18 2005-07-28 Sirna Therapeutics, Inc. RNA interference mediated inhibition of ADAM33 gene expression using short interfering nucleic acid (siNA)
US20050196767A1 (en) * 2001-05-18 2005-09-08 Sirna Therapeutics, Inc. RNA interference mediated inhibition of GRB2 associated binding protein (GAB2) gene expression using short interfering nucleic acis (siNA)
US20060019913A1 (en) * 2001-05-18 2006-01-26 Sirna Therapeutics, Inc. RNA interference mediated inhibtion of protein tyrosine phosphatase-1B (PTP-1B) gene expression using short interfering nucleic acid (siNA)
US20050282188A1 (en) * 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US7517864B2 (en) * 2001-05-18 2009-04-14 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20050288242A1 (en) * 2001-05-18 2005-12-29 Sirna Therapeutics, Inc. RNA interference mediated inhibition of RAS gene expression using short interfering nucleic acid (siNA)
US20050159376A1 (en) * 2002-02-20 2005-07-21 Slrna Therapeutics, Inc. RNA interference mediated inhibition 5-alpha reductase and androgen receptor gene expression using short interfering nucleic acid (siNA)
US20040198682A1 (en) * 2001-11-30 2004-10-07 Mcswiggen James RNA interference mediated inhibition of placental growth factor gene expression using short interfering nucleic acid (siNA)
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US20050124566A1 (en) * 2001-05-18 2005-06-09 Sirna Therapeutics, Inc. RNA interference mediated inhibition of myostatin gene expression using short interfering nucleic acid (siNA)
US20050153915A1 (en) * 2001-05-18 2005-07-14 Sirna Therapeutics, Inc. RNA interference mediated inhibition of early growth response gene expression using short interfering nucleic acid (siNA)
US20050124568A1 (en) * 2001-05-18 2005-06-09 Sirna Therapeutics, Inc. RNA interference mediated inhibition of acetyl-CoA-carboxylase gene expression using short interfering nucleic acid (siNA)
US7109165B2 (en) * 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US20050191638A1 (en) * 2002-02-20 2005-09-01 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US20050136436A1 (en) * 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of G72 and D-amino acid oxidase (DAAO) gene expression using short interfering nucleic acid (siNA)
US20050014172A1 (en) * 2002-02-20 2005-01-20 Ivan Richards RNA interference mediated inhibition of muscarinic cholinergic receptor gene expression using short interfering nucleic acid (siNA)
US20050159382A1 (en) * 2001-05-18 2005-07-21 Sirna Therapeutics, Inc. RNA interference mediated inhibition of polycomb group protein EZH2 gene expression using short interfering nucleic acid (siNA)
US20050233344A1 (en) * 2001-05-18 2005-10-20 Sirna Therapeutics, Inc. RNA interference mediated inhibition of platelet derived growth factor (PDGF) and platelet derived growth factor receptor (PDGFR) gene expression using short interfering nucleic acid (siNA)
US20030175950A1 (en) * 2001-05-29 2003-09-18 Mcswiggen James A. RNA interference mediated inhibition of HIV gene expression using short interfering RNA
US20050137155A1 (en) * 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease using short interfering nucleic acid (siNA)
US20080188430A1 (en) * 2001-05-18 2008-08-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of hypoxia inducible factor 1 (HIF1) gene expression using short interfering nucleic acid (siNA)
US20050196765A1 (en) * 2001-05-18 2005-09-08 Sirna Therapeutics, Inc. RNA interference mediated inhibition of checkpoint Kinase-1 (CHK-1) gene expression using short interfering nucleic acid (siNA)
US20050222066A1 (en) * 2001-05-18 2005-10-06 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20050182009A1 (en) * 2001-05-18 2005-08-18 Sirna Therapeutics, Inc. RNA interference mediated inhibition of NF-Kappa B / REL-A gene expression using short interfering nucleic acid (siNA)
US20050187174A1 (en) * 2001-05-18 2005-08-25 Sirna Therapeutics, Inc. RNA interference mediated inhibition of intercellular adhesion molecule (ICAM) gene expression using short interfering nucleic acid (siNA)
US20050054596A1 (en) * 2001-11-30 2005-03-10 Mcswiggen James RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
JP4358521B2 (ja) 2001-05-18 2009-11-04 サーナ・セラピューティクス・インコーポレイテッド 細胞デリバリーのためのコンジュゲートおよび組成物
US20030170891A1 (en) * 2001-06-06 2003-09-11 Mcswiggen James A. RNA interference mediated inhibition of epidermal growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20050203040A1 (en) * 2001-05-18 2005-09-15 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular cell adhesion molecule (VCAM) gene expression using short interfering nucleic acid (siNA)
US20070270579A1 (en) * 2001-05-18 2007-11-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US20050164967A1 (en) * 2001-05-18 2005-07-28 Sirna Therapeutics, Inc. RNA interference mediated inhibition of platelet-derived endothelial cell growth factor (ECGF1) gene expression using short interfering nucleic acid (siNA)
US20050209180A1 (en) * 2001-05-18 2005-09-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of hepatitis C virus (HCV) expression using short interfering nucleic acid (siNA)
US20050182006A1 (en) * 2001-05-18 2005-08-18 Sirna Therapeutics, Inc RNA interference mediated inhibition of protein kinase C alpha (PKC-alpha) gene expression using short interfering nucleic acid (siNA)
US20050119212A1 (en) * 2001-05-18 2005-06-02 Sirna Therapeutics, Inc. RNA interference mediated inhibition of FAS and FASL gene expression using short interfering nucleic acid (siNA)
US20040019001A1 (en) * 2002-02-20 2004-01-29 Mcswiggen James A. RNA interference mediated inhibition of protein typrosine phosphatase-1B (PTP-1B) gene expression using short interfering RNA
US20080161256A1 (en) * 2001-05-18 2008-07-03 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (siNA)
US20050182007A1 (en) * 2001-05-18 2005-08-18 Sirna Therapeutics, Inc. RNA interference mediated inhibition of interleukin and interleukin receptor gene expression using short interfering nucleic acid (SINA)
US20050176664A1 (en) * 2001-05-18 2005-08-11 Sirna Therapeutics, Inc. RNA interference mediated inhibition of cholinergic muscarinic receptor (CHRM3) gene expression using short interfering nucleic acid (siNA)
US20050143333A1 (en) * 2001-05-18 2005-06-30 Sirna Therapeutics, Inc. RNA interference mediated inhibition of interleukin and interleukin receptor gene expression using short interfering nucleic acid (SINA)
US20050233997A1 (en) * 2001-05-18 2005-10-20 Sirna Therapeutics, Inc. RNA interference mediated inhibition of matrix metalloproteinase 13 (MMP13) gene expression using short interfering nucleic acid (siNA)
EP1390472A4 (de) * 2001-05-29 2004-11-17 Sirna Therapeutics Inc Nukleinsäurebehandlung von krankheiten oder leiden im zusammenhang mit ras-, her2- und hiv-niveaus
US20030140362A1 (en) * 2001-06-08 2003-07-24 Dennis Macejak In vivo models for screening inhibitors of hepatitis B virus
US7205399B1 (en) 2001-07-06 2007-04-17 Sirna Therapeutics, Inc. Methods and reagents for oligonucleotide synthesis
US20030148928A1 (en) * 2001-07-20 2003-08-07 Leonid Beigelman Enzymatic nucleic acid peptide conjugates
US20070203333A1 (en) * 2001-11-30 2007-08-30 Mcswiggen James RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20050075304A1 (en) * 2001-11-30 2005-04-07 Mcswiggen James RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20040138163A1 (en) * 2002-05-29 2004-07-15 Mcswiggen James RNA interference mediated inhibition of vascular edothelial growth factor and vascular edothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20030219775A1 (en) * 2001-12-14 2003-11-27 Ward David C. Nucleic acid diagnostic reagents and methods for detecting nucleic acids, polynucleotides and oligonucleotides
ES2405405T3 (es) 2001-12-17 2013-05-31 Corixa Corporation Composiciones y procedimientos para la terapia y el diagnóstico de enfermedad inflamatoria del intestino
US7071311B2 (en) * 2002-02-13 2006-07-04 Sirna Therapeutics, Inc. Antibodies having specificity for 2′-C-allyl nucleic acids
US20050042632A1 (en) * 2002-02-13 2005-02-24 Sirna Therapeutics, Inc. Antibodies having specificity for nucleic acids
US20050137153A1 (en) * 2002-02-20 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of alpha-1 antitrypsin (AAT) gene expression using short interfering nucleic acid (siNA)
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
WO2003106476A1 (en) * 2002-02-20 2003-12-24 Sirna Therapeutics, Inc Nucleic acid mediated inhibition of enterococcus infection and cytolysin toxin activity
US20050096284A1 (en) * 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US20050222064A1 (en) * 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
EP1432724A4 (de) 2002-02-20 2006-02-01 Sirna Therapeutics Inc Durch rna-interferenz vermittelte inhibierung von map-kinase-genen
US20030195164A1 (en) * 2002-04-16 2003-10-16 Veli-Matti Kahari Novel ribozyme and its use
US20040018520A1 (en) * 2002-04-22 2004-01-29 James Thompson Trans-splicing enzymatic nucleic acid mediated biopharmaceutical and protein
US20030224435A1 (en) * 2002-05-16 2003-12-04 Scott Seiwert Detection of abused substances and their metabolites using nucleic acid sensor molecules
US7767803B2 (en) 2002-06-18 2010-08-03 Archemix Corp. Stabilized aptamers to PSMA and their use as prostate cancer therapeutics
US6989442B2 (en) * 2002-07-12 2006-01-24 Sirna Therapeutics, Inc. Deprotection and purification of oligonucleotides and their derivatives
US7655790B2 (en) * 2002-07-12 2010-02-02 Sirna Therapeutics, Inc. Deprotection and purification of oligonucleotides and their derivatives
AU2003261264B2 (en) * 2002-07-25 2008-12-11 Archemix Corp. Regulated aptamer therapeutics
EP1857547B2 (de) 2002-08-05 2020-12-02 Silence Therapeutics GmbH Weitere neue Formen von interferierenden RNA-Molekülen
US7956176B2 (en) 2002-09-05 2011-06-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US20080260744A1 (en) 2002-09-09 2008-10-23 Omeros Corporation G protein coupled receptors and uses thereof
US9303262B2 (en) 2002-09-17 2016-04-05 Archemix Llc Methods for identifying aptamer regulators
AU2003273336A1 (en) * 2002-09-18 2004-04-08 Isis Pharmaceuticals, Inc. Efficient reduction of target rna's by single- and double-stranded oligomeric compounds
EP1556402B1 (de) 2002-09-25 2011-06-22 University of Massachusetts Abstellen von genen in vivo durch chemischmodifizierte und stabile sirna
CA2504929C (en) 2002-11-05 2014-07-22 Charles Allerson Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
US10100316B2 (en) * 2002-11-21 2018-10-16 Archemix Llc Aptamers comprising CPG motifs
US8039443B2 (en) * 2002-11-21 2011-10-18 Archemix Corporation Stabilized aptamers to platelet derived growth factor and their use as oncology therapeutics
WO2004047742A2 (en) * 2002-11-21 2004-06-10 Archemix Corporation Multivalent aptamer therapeutics with improved pharmacodynamic properties and methods of making and using the same
US20050124565A1 (en) * 2002-11-21 2005-06-09 Diener John L. Stabilized aptamers to platelet derived growth factor and their use as oncology therapeutics
US8853376B2 (en) 2002-11-21 2014-10-07 Archemix Llc Stabilized aptamers to platelet derived growth factor and their use as oncology therapeutics
US20050037394A1 (en) * 2002-12-03 2005-02-17 Keefe Anthony D. Method for in vitro selection of 2'-substituted nucleic acids
US20040197804A1 (en) * 2002-12-03 2004-10-07 Keefe Anthony D. Method for in vitro selection of 2'-substituted nucleic acids
WO2004055198A2 (en) * 2002-12-12 2004-07-01 Chiron Corporation Device and method for in-line blood testing using biochips
US20030232400A1 (en) * 2002-12-20 2003-12-18 Susan Radka Methods of screening subjects for expression of soluble receptors of vascular endothelial growth factor (VEGF) for use in managing treatment and determining prognostic outcome
US20040231231A1 (en) * 2002-12-20 2004-11-25 Cataldo Dominic A. Use of colloidal clays for sustained release of active ingredients
US20050085435A1 (en) * 2003-01-02 2005-04-21 Niles Edward G. Novel inhibitors of poxvirus replication
WO2004089294A2 (en) 2003-04-04 2004-10-21 Incyte Corporation Compositions, methods and kits relating to her-2 cleavage
WO2004093788A2 (en) * 2003-04-17 2004-11-04 The Trustees Of Columbia University In The City Ofnew York Desmoglein 4 is a novel gene involved in hair growth
JP2007525177A (ja) 2003-04-21 2007-09-06 アーケミックス コーポレイション 血小板由来増殖因子に対する安定化アプタマーおよび腫瘍治療剤としてのそれらの使用
US20050250106A1 (en) * 2003-04-24 2005-11-10 David Epstein Gene knock-down by intracellular expression of aptamers
EP3222294A1 (de) 2003-04-30 2017-09-27 Sirna Therapeutics, Inc. Konjugate und zusammensetzungen zur zellulären verabreichung
WO2005078848A2 (en) * 2004-02-11 2005-08-25 University Of Tennessee Research Foundation Inhibition of tumor growth and invasion by anti-matrix metalloproteinase dnazymes
US7803931B2 (en) 2004-02-12 2010-09-28 Archemix Corp. Aptamer therapeutics useful in the treatment of complement-related disorders
PT3385384T (pt) 2004-02-12 2020-06-30 Archemix Llc Terapêuticas com aptâmeros úteis no tratamento de desordens relacionadas com o complemento
US20060193821A1 (en) * 2004-03-05 2006-08-31 Diener John L Aptamers to the human IL-12 cytokine family and their use as autoimmune disease therapeutics
CA2557633A1 (en) * 2004-03-05 2005-09-22 Archemix Corp. Aptamers to the human il-12 cytokine family and their use as autoimmune disease therapeutics
US8569474B2 (en) 2004-03-09 2013-10-29 Isis Pharmaceuticals, Inc. Double stranded constructs comprising one or more short strands hybridized to a longer strand
EP1735009A4 (de) 2004-03-12 2011-03-30 Alnylam Pharmaceuticals Inc Irna mittel targeting vegf
US20080214489A1 (en) * 2004-04-19 2008-09-04 Anthony Dominic Keefe Aptamer-mediated intracellular delivery of oligonucleotides
WO2005105157A2 (en) 2004-04-23 2005-11-10 The Trustees Of Columbia University In The City Ofnew York INHIBITION OF HAIRLESS PROTEIN mRNA
US7579450B2 (en) 2004-04-26 2009-08-25 Archemix Corp. Nucleic acid ligands specific to immunoglobulin E and their use as atopic disease therapeutics
US20060040882A1 (en) 2004-05-04 2006-02-23 Lishan Chen Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
US10508277B2 (en) 2004-05-24 2019-12-17 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
CA2574572A1 (en) 2004-07-19 2006-10-26 Baylor College Of Medicine Modulation of cytokine signaling regulators and applications for immunotherapy
AU2005272920B2 (en) 2004-08-10 2011-05-12 Institute For Multiple Myeloma And Bone Cancer Research Methods of regulating differentiation and treating of multiple myeloma
US7566701B2 (en) 2004-09-07 2009-07-28 Archemix Corp. Aptamers to von Willebrand Factor and their use as thrombotic disease therapeutics
WO2006029258A2 (en) 2004-09-07 2006-03-16 Archemix Corp. Aptamer medicinal chemistry
EP1789096A4 (de) 2004-09-07 2009-07-08 Archemix Corp Aptamere für den von-willebrand-faktor und ihre verwendung als therapeutika für thrombotische erkrankungen
US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
EP3034089A1 (de) 2004-11-02 2016-06-22 Archemix LLC Stabilisierte aptamere für einen aus plättchen gewonnenen wachstumsfaktor und deren verwendung als onkologietherapeutika
ES2401285T3 (es) 2004-12-16 2013-04-18 The Regents Of The University Of California Fármacos con el pulmón como diana
AU2006220621A1 (en) * 2005-03-07 2006-09-14 Archemix Corp. Stabilized aptamers to PSMA and their use as prostate cancer therapeutics
EP1866414B9 (de) 2005-03-31 2012-10-03 Calando Pharmaceuticals, Inc. Inhibitoren der untereinheit 2 der ribonukleotidreduktase und ihre verwendungen
US20090075342A1 (en) * 2005-04-26 2009-03-19 Sharon Cload Metabolic profile directed aptamer medicinal chemistry
US7868159B2 (en) 2005-06-23 2011-01-11 Baylor College Of Medicine Modulation of negative immune regulators and applications for immunotherapy
EP2062980B1 (de) 2005-06-28 2011-08-31 Medtronic, Inc. Verfahren und Nukleotid-Sequenzen, die bevorzugt die Expression eines mutierten Huntingtin-Genes unterdrücken
US8101385B2 (en) 2005-06-30 2012-01-24 Archemix Corp. Materials and methods for the generation of transcripts comprising modified nucleotides
MX2007016561A (es) 2005-06-30 2008-03-10 Archemix Corp Materiales y metodos para la generacion de transcriptos de acido nucleico completamente modificados en la posicion 2.
US20090176725A1 (en) * 2005-08-17 2009-07-09 Sirna Therapeutics Inc. Chemically modified short interfering nucleic acid molecules that mediate rna interference
FI20055712A0 (fi) * 2005-12-29 2005-12-29 Wallac Oy Makrosykliset oligonukleotiidien leimausreagenssit ja niistä johdetut konjugaatit
JP5829373B2 (ja) 2006-01-27 2015-12-09 バイオジェン・エムエイ・インコーポレイテッドBiogen MA Inc. Nogoレセプターアンタゴニスト
US7922000B2 (en) * 2006-02-15 2011-04-12 Miraial Co., Ltd. Thin plate container with a stack of removable loading trays
HUE026496T2 (en) 2006-03-08 2016-06-28 Archemix Llc Complement-binding aptamers and anti-C5 agents for treating eye diseases
EP2527354A1 (de) 2006-03-31 2012-11-28 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und Verfahren zur Hemmung der Expression des Eg5-Gens
JP5524610B2 (ja) 2006-04-07 2014-06-18 ザ リサーチ ファウンデーション オブ ステイト ユニバーシティー オブ ニューヨーク トランスコバラミン受容体ポリペプチド、核酸およびその調節因子、ならびに細胞の増殖の調節ならびにガンおよびコバラミン欠損症の処置に使用する関連方法
US8158782B2 (en) 2006-05-05 2012-04-17 Wallac Oy Biomolecule labeling reactants based on azacycloalkanes and conjugates derived thereof
US20070275923A1 (en) * 2006-05-25 2007-11-29 Nastech Pharmaceutical Company Inc. CATIONIC PEPTIDES FOR siRNA INTRACELLULAR DELIVERY
US8598333B2 (en) 2006-05-26 2013-12-03 Alnylam Pharmaceuticals, Inc. SiRNA silencing of genes expressed in cancer
JP5248494B2 (ja) 2006-07-11 2013-07-31 ユニバーシティ・オブ・メディシン・アンド・デンティストリー・オブ・ニュージャージー タンパク質、それをコードする核酸および関連する使用方法
CN101557818A (zh) 2006-10-03 2009-10-14 新泽西医科和牙科大学 Atap肽、编码atap肽的核酸以及相关的应用方法
EP2081596A4 (de) 2006-10-06 2010-07-21 Univ Utah Res Found Verfahren zum nachweis von augenkrankheiten und pathologischen zuständen und behandlung dafür
WO2008052774A2 (en) 2006-10-31 2008-05-08 Noxxon Pharma Ag Methods for detection of a single- or double-stranded nucleic acid molecule
US20100129358A1 (en) 2006-12-22 2010-05-27 University Of Utah Research Foundation Method of detecting ocular diseases and pathologic conditions and treatment of same
AU2008242842B2 (en) * 2007-04-17 2014-06-05 Baxter Healthcare Sa Nucleic acid microparticles for pulmonary delivery
JP5475643B2 (ja) 2007-05-04 2014-04-16 マリーナ バイオテック,インコーポレイテッド アミノ酸脂質およびその使用
EP2164989B1 (de) 2007-05-23 2018-01-24 The Trustees Of The University Of Pennsylvania Trägerstoffe als ziele für intrazelluläre arzneistoffzuführung
US8183221B2 (en) 2007-09-05 2012-05-22 Medtronic, Inc. Suppression of SCN9A gene expression and/or function for the treatment of pain
EP2195432B1 (de) 2007-09-24 2015-12-09 Performance Plants, Inc. Pflanzen mit erhöhter biomasse
EP2222344A4 (de) 2007-11-30 2012-11-07 Baylor College Medicine Impfstoffzusammensetzungen mit dendritischen zellen und ihre verwendung
AU2008340355B2 (en) 2007-12-04 2015-01-22 Tekmira Pharmaceuticals Corporation Targeting lipids
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
BRPI0911332A2 (pt) 2008-04-04 2019-09-24 Calando Pharmaceuticals Inc composições e uso de inibidores de epas1
JP5475753B2 (ja) 2008-04-15 2014-04-16 プロチバ バイオセラピューティクス インコーポレイティッド 核酸送達用の脂質製剤
US20110229498A1 (en) 2008-05-08 2011-09-22 The Johns Hopkins University Compositions and methods for modulating an immune response
ATE550024T1 (de) 2008-05-30 2012-04-15 Univ Yale Gezielte oligonucleotid-zusammensetzungen zur modifizierung der genexpression
CA2737146A1 (en) 2008-07-25 2010-01-28 The Regents Of The University Of Colorado Clip inhibitors and methods of modulating immune function
US9249175B2 (en) * 2008-09-05 2016-02-02 The Royal Institute For The Advancement Of Learning/Mcgill University RNA monomers containing O-acetal levulinyl ester groups and their use in RNA microarrays
EP3025727A1 (de) 2008-10-02 2016-06-01 The J. David Gladstone Institutes Verfahren zur behandlung von lebererkrankungen
EP2743265B1 (de) 2008-10-09 2017-03-15 Arbutus Biopharma Corporation Verbesserte Aminolipide und Verfahren zur Freisetzung von Nukleinsäuren
SG196818A1 (en) 2008-10-16 2014-02-13 Marina Biotech Inc Processes and compositions for liposomal and efficient delivery of gene silencing therapeutics
DK3199553T3 (da) 2008-10-29 2019-07-22 Circular Commitment Company Fremgangsmåder og midler til diagnosticering og behandling af hepatocellulært carcinom
EP3207944B1 (de) 2008-11-10 2020-01-15 Arbutus Biopharma Corporation Neuartige lipide und zusammensetzungen zur verabreichung von therapeutika
WO2010088537A2 (en) 2009-01-29 2010-08-05 Alnylam Pharmaceuticals, Inc. Improved lipid formulation
US20100215660A1 (en) 2009-02-23 2010-08-26 Sarwar Hashmi Kruppel-like factors and fat regulation
ES2628144T3 (es) 2009-04-03 2017-08-01 Dicerna Pharmaceuticals, Inc. Métodos y composiciones para la inhibición específica de KRAS por RNA bicatenario asimétrico
EP2756845B1 (de) 2009-04-03 2017-03-15 Dicerna Pharmaceuticals, Inc. Verfahren und Zusammensetzungen für spezifische KRAS-Hemmung mit asymmetrischer Doppelstrang-RNA
JP2012523225A (ja) 2009-04-10 2012-10-04 アソシアシオン・アンスティテュ・ドゥ・ミオロジー 疾患の処置のためのトリシクロ−dnaアンチセンスオリゴヌクレオチド、組成物及び方法
WO2010120266A1 (en) 2009-04-13 2010-10-21 Inserm, Institut National De La Sante Et De La Recherche Medicale Hpv particles and uses thereof
AU2010245933B2 (en) 2009-05-05 2016-06-16 Arbutus Biopharma Corporation Methods of delivering oligonucleotides to immune cells
NZ596186A (en) 2009-05-05 2014-03-28 Alnylam Pharmaceuticals Inc Lipid compositions
CN102985548B (zh) 2009-05-16 2016-10-26 崔坤元 用于递送治疗分子的包含阳离子两亲物和辅脂质的组合物
HUE038796T2 (hu) 2009-06-10 2018-11-28 Arbutus Biopharma Corp Továbbfejlesztett lipid készítmény
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
WO2011039646A2 (en) 2009-09-30 2011-04-07 Inserm (Institut National De La Sante Et De La Recherche Medicale) Papilloma virus -like particles for targeted gene delivery
US8450090B2 (en) 2009-10-06 2013-05-28 The Regents Of The University Of Colorado, A Body Corporate Compositions and methods for promoting fatty acid production in plants
US20120270930A1 (en) 2009-10-29 2012-10-25 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Methods and compositions for dysferlin exon-skipping
WO2011062962A2 (en) 2009-11-17 2011-05-26 The Trustees Of The University Of Pennsylvania Smndelta7 degron: novel compositions and methods of use
WO2011071860A2 (en) 2009-12-07 2011-06-16 Alnylam Pharmaceuticals, Inc. Compositions for nucleic acid delivery
US20130017223A1 (en) 2009-12-18 2013-01-17 The University Of British Columbia Methods and compositions for delivery of nucleic acids
MX2012009318A (es) 2010-02-10 2012-09-07 Novartis Ag Metodos y compuestos para el crecimiento muscular.
JP5914362B2 (ja) 2010-02-11 2016-05-11 ナノストリング テクノロジーズ, インコーポレイテッド 低分子rna分子の検出のための組成物および方法
WO2011119852A1 (en) 2010-03-24 2011-09-29 Rxi Pharmaceuticals Corporation Reduced size self-delivering rnai compounds
EP2550361B1 (de) 2010-03-25 2017-02-08 The J. David Gladstone Institutes Zusammensetzungen und verfahren zur behandlung neurologischer störungen
WO2011120023A1 (en) 2010-03-26 2011-09-29 Marina Biotech, Inc. Nucleic acid compounds for inhibiting survivin gene expression uses thereof
WO2011133584A2 (en) 2010-04-19 2011-10-27 Marina Biotech, Inc. Nucleic acid compounds for inhibiting hras gene expression and uses thereof
KR20200047642A (ko) 2010-04-23 2020-05-07 유니버시티 오브 플로리다 리서치 파운데이션, 인크. 레베르 선천성 흑내장-1(lca1)의 치료를 위한 재조합 아데노-관련된 바이러스-구아닐레이트 사이클라제 조성물 및 방법
WO2011139710A1 (en) 2010-04-26 2011-11-10 Marina Biotech, Inc. Nucleic acid compounds with conformationally restricted monomers and uses thereof
WO2011139843A2 (en) 2010-04-28 2011-11-10 Marina Biotech, Inc. Multi-sirna compositions for reducing gene expression
CA2804214C (en) 2010-07-06 2022-04-12 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of beta-catenin by double-stranded rna
US10064885B2 (en) 2010-07-09 2018-09-04 Massachusetts Institute Of Technology Metabolic gene, enzyme, and flux targets for cancer therapy
WO2012016188A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
WO2012016184A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
EP3372684B1 (de) 2010-08-24 2020-10-07 Sirna Therapeutics, Inc. Einsträngige rnai-mittel mit einem internen nukleinsäurefreien spacer
WO2012044992A2 (en) 2010-09-30 2012-04-05 Agency For Science, Technology And Research (A*Star) Methods and reagents for detection and treatment of esophageal metaplasia
EP3327125B1 (de) 2010-10-29 2020-08-05 Sirna Therapeutics, Inc. Rna-interferenz-vermittelte inhibition von genexpression unter verwendung kurzer interferierender nukleinsäure (sina)
US9072766B2 (en) 2010-11-18 2015-07-07 Beth Israel Deaconess Medical Center, Inc. Methods of treating obesity by inhibiting nicotinamide N-methyl transferase (NNMT)
WO2012097093A2 (en) 2011-01-11 2012-07-19 Fate Therapeutics, Inc. Novel wnt compositions and therapeutic uses of such compositions
DK2663548T3 (en) 2011-01-11 2017-07-24 Alnylam Pharmaceuticals Inc PEGYLED LIPIDS AND THEIR USE FOR PHARMACEUTICAL SUPPLY
EP2753631A1 (de) 2011-09-07 2014-07-16 Marina Biotech, Inc. Synthese und verwendung von nukleinsäureverbindungen mit konformationseingeschränkten monomeren
US9701623B2 (en) 2011-09-27 2017-07-11 Alnylam Pharmaceuticals, Inc. Di-aliphatic substituted pegylated lipids
RS57645B1 (sr) 2011-10-14 2018-11-30 Hoffmann La Roche Anti-htra1 antitela i načini primene
EP2766501B1 (de) 2011-10-14 2020-01-01 Accugenomics, Inc. Quantitative Nukleinsäureamplifikation
WO2013090457A2 (en) 2011-12-12 2013-06-20 Oncoimmunin Inc. In vivo delivery of oligonucleotides
EP2797948B1 (de) 2011-12-27 2019-02-20 The J. David Gladstone Institutes Zusammensetzungen zur verwendung in der regulierung des glukosemetabolismus
US9700639B2 (en) 2012-02-07 2017-07-11 Aura Biosciences, Inc. Virion-derived nanospheres for selective delivery of therapeutic and diagnostic agents to cancer cells
US9352042B2 (en) 2012-02-24 2016-05-31 Protiva Biotherapeutics, Inc. Trialkyl cationic lipids and methods of use thereof
EP3553181A1 (de) 2012-05-25 2019-10-16 Accugenomics, Inc. Nukleinsäureamplifikation und anwendung davon
ES2785274T3 (es) 2012-07-05 2020-10-06 Univ Pennsylvania La RNPsn U1 regula la expresión génica y modula la oncogenicidad
KR101520383B1 (ko) 2012-08-02 2015-05-15 에이비온 주식회사 Hpv 감염과 관련된 암의 치료용 조성물
WO2014039513A2 (en) 2012-09-04 2014-03-13 The Trustees Of The University Of Pennsylvania Inhibition of diacylglycerol kinase to augment adoptive t cell transfer
KR20150090072A (ko) 2012-10-23 2015-08-05 카리스 라이프 사이언스 스위스 홀딩스 게엠베하 압타머 및 이의 용도
US10942184B2 (en) 2012-10-23 2021-03-09 Caris Science, Inc. Aptamers and uses thereof
WO2014100434A1 (en) 2012-12-19 2014-06-26 Caris Science, Inc. Compositions and methods for aptamer screening
CN105189752B (zh) 2013-03-08 2021-01-26 耶鲁大学 用于减少皮肤色素沉着的组合物和方法
BR112015023705A8 (pt) 2013-03-15 2020-03-17 Univ California compostos, composições farmacêuticas, usos de um composto, e método para síntese do composto da fórmula (ia)
EP4012031A1 (de) 2013-07-03 2022-06-15 Dicerna Pharmaceuticals, Inc. Verfahren und zusammensetzungen zur spezifischen alpha-1-trypsin-hemmung durch doppelsträngige rna
CA3177846A1 (en) 2013-07-11 2015-01-15 Alnylam Pharmaceuticals, Inc. Oligonucleotide-ligand conjugates and process for their preparation
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
AU2014312211A1 (en) 2013-08-28 2016-03-10 Caris Science, Inc. Oligonucleotide probes and uses thereof
DK3046583T3 (da) 2013-09-18 2019-05-20 Aura Biosciences Inc Viruslignende partikelkonjugater til behandling af tumorer
US9974788B2 (en) 2013-09-26 2018-05-22 Beth Israel Deaconess Medical Center, Inc. Inhibition of SGK1 in the treatment of heart conditions
US9365610B2 (en) 2013-11-18 2016-06-14 Arcturus Therapeutics, Inc. Asymmetric ionizable cationic lipid for RNA delivery
WO2015074010A2 (en) 2013-11-18 2015-05-21 Beth Israel Deaconess Medical Center, Inc. Compositions and methods for cardiac regeneration
EP3071547A1 (de) 2013-11-18 2016-09-28 Arcturus Therapeutics, Inc. Ionisierbares kationisches lipid für rna-freisetzung
AR093574A1 (es) 2013-11-21 2015-06-10 Consejo Nac De Investig Cientificas Y Tecn (Conicet) Aptameros contra la proteina basica de mielina como agentes neuroprotectores
CN106061488B (zh) 2013-12-02 2021-04-09 菲奥医药公司 癌症的免疫治疗
ES2936812T3 (es) 2013-12-20 2023-03-22 Acetylon Pharmaceuticals Inc Biomarcadores de histona desacetilasa 6 (HDAC6) en mieloma múltiple
EP3581654B1 (de) 2013-12-27 2021-03-31 Dicerna Pharmaceuticals, Inc. Verfahren und zusammensetzungen für die spezifische hemmung von glycolatoxidase (hao1) durch doppelstrang-rna
US9458464B2 (en) 2014-06-23 2016-10-04 The Johns Hopkins University Treatment of neuropathic pain
JP6789124B2 (ja) 2014-07-03 2020-11-25 イエール ユニバーシティ 腫瘍形成を抑制するディコップ2(Dickkopf2)(Dkk2)阻害
CA2956224A1 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
KR20230145550A (ko) 2014-09-15 2023-10-17 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 뉴클레오타이드 유사체
AU2015330670B2 (en) 2014-10-10 2022-01-06 Novo Nordisk Health Care Ag Therapeutic inhibition of lactate dehydrogenase and agents therefor
WO2016061131A1 (en) 2014-10-14 2016-04-21 The J. David Gladstone Institutes Compositions and methods for reactivating latent immunodeficiency virus
RS64078B1 (sr) 2014-11-18 2023-04-28 Arcturus Therapeutics Inc Jonizujući katjonski lipid za isporuku rnk
US20170355755A1 (en) 2014-11-21 2017-12-14 Yale University Compositions and methods for modulating salm5 and hvem
WO2016100401A1 (en) 2014-12-15 2016-06-23 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
CN107208096B (zh) 2014-12-18 2021-02-12 综合基因技术公司 基于crispr的组合物和使用方法
MA41629A (fr) 2015-03-04 2018-01-09 Center For Human Reproduction Compositions et méthodes d'utilisation de l'hormone anti-müllérienne pour le traitement de l'infertilité
AU2016229076B2 (en) 2015-03-09 2022-01-20 Caris Science, Inc. Oligonucleotide probes and uses thereof
CA2991045A1 (en) 2015-06-29 2017-01-05 Caris Science, Inc. Therapeutic oligonucleotides binding c1q
US10941176B2 (en) 2015-07-28 2021-03-09 Caris Science, Inc. Therapeutic oligonucleotides
WO2017023861A1 (en) 2015-08-03 2017-02-09 The Regents Of The University Of California Compositions and methods for modulating abhd2 activity
PE20181009A1 (es) 2015-10-30 2018-06-26 Genentech Inc ANTICUERPOS ANTI-HtrA1 Y METODOS DE USO DE LOS MISMOS
US11207339B2 (en) 2015-10-30 2021-12-28 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Targeted cancer therapy
CA3018066A1 (en) 2016-03-18 2017-09-21 Caris Science, Inc. Oligonucleotide probes and uses thereof
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA45468A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques myc et utilisations
MA45470A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques kras et leurs utilisations
MA45471A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques de phosphatidylinositol-3-kinase et leurs utilisations
MA45349A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques egfr et leurs utilisations
MA45469A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques de bêta-caténine et leurs utilisations
US20190119364A1 (en) 2016-04-29 2019-04-25 The Regents Of The University Of Colorado, A Body Corporate Compounds and compositions useful for treating metabolic syndrome, and methods using same
AU2017271579B2 (en) 2016-05-25 2023-10-19 Caris Science, Inc. Oligonucleotide probes and uses thereof
CN109804066A (zh) 2016-08-09 2019-05-24 哈佛大学的校长及成员们 可编程cas9-重组酶融合蛋白及其用途
WO2018035141A1 (en) 2016-08-16 2018-02-22 Bluebird Bio, Inc. Il-10 receptor alpha homing endonuclease variants, compositions, and methods of use
EP3504335A4 (de) 2016-08-23 2020-04-15 Bluebird Bio, Inc. Tim3-homing-endonuklease-varianten, zusammensetzungen und verfahren zur verwendung
KR102451510B1 (ko) 2016-09-08 2022-10-07 2세븐티 바이오, 인코포레이티드 Pd-1 호밍 엔도뉴클레아제 변이체, 조성물 및 사용 방법
US11242542B2 (en) 2016-10-07 2022-02-08 Integrated Dna Technologies, Inc. S. pyogenes Cas9 mutant genes and polypeptides encoded by same
EP3523427A4 (de) 2016-10-07 2020-09-02 Integrated DNA Technologies, Inc. S.-pyogenes-cas9-mutantengene und dadurch codierte polypeptide
MA46862A (fr) 2016-11-17 2019-09-25 Bluebird Bio Inc Convertisseur de signaux tgf béta
AU2017364084A1 (en) 2016-11-22 2019-05-16 Integrated Dna Technologies, Inc. CRISPR/Cpf1 systems and methods
WO2018111947A1 (en) 2016-12-12 2018-06-21 Integrated Dna Technologies, Inc. Genome editing enhancement
US10383952B2 (en) 2016-12-21 2019-08-20 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2018119163A1 (en) 2016-12-21 2018-06-28 Payne Joseph E Ionizable cationic lipid for rna delivery
US10526284B2 (en) 2016-12-21 2020-01-07 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2018119359A1 (en) 2016-12-23 2018-06-28 President And Fellows Of Harvard College Editing of ccr5 receptor gene to protect against hiv infection
CA3049424A1 (en) 2017-01-06 2018-07-12 Avidity Biosciences Llc Nucleic acid-polypeptide compositions and methods of inducing exon skipping
CA3053499A1 (en) 2017-02-15 2018-08-23 Bluebird Bio, Inc. Donor repair templates multiplex genome editing
US11898179B2 (en) 2017-03-09 2024-02-13 President And Fellows Of Harvard College Suppression of pain by gene editing
GB201711809D0 (en) 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
US11732274B2 (en) 2017-07-28 2023-08-22 President And Fellows Of Harvard College Methods and compositions for evolving base editors using phage-assisted continuous evolution (PACE)
EP3676376A2 (de) 2017-08-30 2020-07-08 President and Fellows of Harvard College Hocheffiziente baseneditoren mit gam
WO2019046698A1 (en) 2017-09-01 2019-03-07 Thomas Jefferson University COMPOSITIONS AND METHODS FOR MYC GENE MESSENGER RNA INHIBITORS
MX2020003596A (es) 2017-10-04 2020-07-22 Avidity Biosciences Inc Composiciones de acido nucleico-polipeptido y usos de las mismos.
US11795443B2 (en) 2017-10-16 2023-10-24 The Broad Institute, Inc. Uses of adenosine base editors
KR102527941B1 (ko) 2017-12-06 2023-05-02 어비디티 바이오사이언시스 인크. 근위축증 및 근긴장성 이영양증을 치료하는 조성물 및 방법
US11713446B2 (en) 2018-01-08 2023-08-01 Iovance Biotherapeutics, Inc. Processes for generating TIL products enriched for tumor antigen-specific T-cells
WO2019136459A1 (en) 2018-01-08 2019-07-11 Iovance Biotherapeutics, Inc. Processes for generating til products enriched for tumor antigen-specific t-cells
BR112020013848A2 (pt) 2018-01-08 2020-12-01 Iovance Biotherapeutics, Inc. métodos para expandir linfócitos infiltrantes de tumor e para tratar um indivíduo com câncer, população de linfócitos infiltrantes de tumor, e, método para avaliar fatores de transcrição
CN112424352A (zh) 2018-05-11 2021-02-26 阿尔法阿诺麦里克公司 包括7′-5′-α-异头双环糖核苷的寡核苷酸缀合物
US11685906B2 (en) 2018-12-06 2023-06-27 Arcturus Therapeutics, Inc. Compositions and methods for treating ornithine transcarbamylase deficiency
WO2020191248A1 (en) 2019-03-19 2020-09-24 The Broad Institute, Inc. Method and compositions for editing nucleotide sequences
US20220204975A1 (en) 2019-04-12 2022-06-30 President And Fellows Of Harvard College System for genome editing
CA3142283A1 (en) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
MX2022001720A (es) 2019-08-14 2022-03-11 Acuitas Therapeutics Inc Nanoparticulas lipidicas mejoradas para el suministro de acidos nucleicos.
WO2021188390A1 (en) 2020-03-19 2021-09-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
AU2021241682A1 (en) 2020-03-27 2022-10-13 Avidity Biosciences, Inc. Compositions and methods of treating muscle dystrophy
JP2023525304A (ja) 2020-05-08 2023-06-15 ザ ブロード インスティテュート,インコーポレーテッド 標的二本鎖ヌクレオチド配列の両鎖同時編集のための方法および組成物
WO2022097157A2 (en) 2020-11-09 2022-05-12 1E Therapeutics, Ltd. Catalytic sequence based methods of treating or preventing bacterial infections
KR20230132472A (ko) 2020-12-18 2023-09-15 예다 리서치 앤드 디벨럽먼트 캄파니 리미티드 Chd2 반수체기능부전의 치료에 사용하기 위한 조성물및 이를 확인하는 방법
WO2022144883A2 (en) 2020-12-28 2022-07-07 1E Therapeutics, Ltd. P21 mrna targeting dnazymes
AU2021411103A1 (en) 2020-12-28 2023-07-13 1E Therapeutics, Ltd. P21 mrna target areas for silencing
CA3207359A1 (en) 2021-02-05 2022-08-11 Cecile Chartier-Courtaud Adjuvant therapy for cancer
KR20240009393A (ko) 2021-03-31 2024-01-22 엔트라다 테라퓨틱스, 인크. 사이클릭 세포 침투 펩티드
CN117242173A (zh) 2021-05-03 2023-12-15 安斯泰来再生医药协会 产生成熟角膜内皮细胞的方法
WO2022240760A2 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. COMPOSITIONS AND METHODS FOR MODULATING mRNA SPLICING
WO2022240721A1 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Compositions and methods for modulating interferon regulatory factor-5 (irf-5) activity
AU2022271873A1 (en) 2021-05-10 2024-01-04 Entrada Therapeutics, Inc. Compositions and methods for intracellular therapeutics
KR20240021235A (ko) 2021-06-14 2024-02-16 2세븐티 바이오, 인코포레이티드 단일 가닥 rna 정제 방법
WO2022271955A1 (en) 2021-06-23 2022-12-29 Musc Foundation For Research Development Novel targeted shrna nanoparticles for cancer therapy
IL309001A (en) 2021-06-23 2024-02-01 Entrada Therapeutics Inc Antisense compounds and methods for targeting CUG repeats
AU2022324471A1 (en) 2021-08-05 2024-02-15 Sanegene Bio Usa Inc. 1'-alkyl modified ribose derivatives and methods of use
CA3231330A1 (en) 2021-09-16 2023-03-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
AU2022352676A1 (en) 2021-09-22 2024-03-14 Sanegene Bio Usa Inc. 2'-alkyl or 3'- alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides
US20230227480A1 (en) 2021-10-05 2023-07-20 Sanegene Bio Usa Inc. Polyhydroxylated cyclopentane derivatives and methods of use
WO2023144798A1 (en) 2022-01-31 2023-08-03 Genevant Sciences Gmbh Ionizable cationic lipids for lipid nanoparticles
WO2023144792A1 (en) 2022-01-31 2023-08-03 Genevant Sciences Gmbh Poly(alkyloxazoline)-lipid conjugates and lipid particles containing same
WO2023164464A1 (en) 2022-02-22 2023-08-31 Sanegene Bio Usa Inc. 5'-modified carbocyclic ribonucleotide derivatives and methods of use
WO2024015796A1 (en) 2022-07-11 2024-01-18 Sanegene Bio Usa Inc. Optimized 2'- modified ribose derivatives and methods of use
WO2024026474A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4987071A (en) * 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
ATE115999T1 (de) * 1987-12-15 1995-01-15 Gene Shears Pty Ltd Ribozyme.
ES2090049T3 (es) * 1989-03-16 1996-10-16 Boehringer Ingelheim Int Unidades geneticas para la inhibicion de la funcion de arn.
US5149796A (en) * 1989-08-31 1992-09-22 City Of Hope Chimeric DNA-RNA catalytic sequences
JP3058686B2 (ja) * 1989-08-31 2000-07-04 シティ・オブ・ホープ キメラdna―rna触媒活性配列
KR930701590A (ko) * 1990-06-19 1993-06-12 알프레드 퍼나트 엔도누클레아제
AU649074B2 (en) * 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
DE4216134A1 (de) * 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US5817635A (en) * 1993-08-09 1998-10-06 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes

Also Published As

Publication number Publication date
JPH06501842A (ja) 1994-03-03
AU8613991A (en) 1992-05-20
JP3257675B2 (ja) 2002-02-18
ES2061416T3 (es) 1997-03-01
AU649074B2 (en) 1994-05-12
WO1992007065A1 (en) 1992-04-30
JP2002101893A (ja) 2002-04-09
CA2093664C (en) 2003-07-29
US5672695A (en) 1997-09-30
EP0552178A1 (de) 1993-07-28
PL169576B1 (pl) 1996-08-30
US6890908B1 (en) 2005-05-10
ES2061416T1 (es) 1994-12-16
CA2093664A1 (en) 1992-04-13
GR930300130T1 (en) 1993-12-30
US5698687A (en) 1997-12-16
DE552178T1 (de) 1994-02-03
EP0552178B1 (de) 1997-01-02
DE69123979D1 (de) 1997-02-13
ATE147098T1 (de) 1997-01-15

Similar Documents

Publication Publication Date Title
DE69123979T2 (de) Abgeänderte ribozyme
Reich et al. Structure-activity correlations of actinomycins and their derivatives.
MY104069A (en) Therapeutic nucleosides
BR9106743A (pt) Composicao,analogo de nucleotideo,processo para sua preparacao e processo para modular a atividade de rna
ATE37984T1 (de) Pharmazeutische zusammensetzung welche 5acetamido-3,5-dideoxy-d-glycero-dgalactononulosamins|ure cytidin monophosphat enthaelt.
DK35684D0 (da) Antitumor-midler
DE69527942D1 (de) Modifizierte oligonukleotide
EP0305117A3 (en) 2',3'-dideoxy-3'-fluorothymidine and related compounds for the treatment of adenovirus infections
CA2002648A1 (en) 2'-halomethylidene, 2'-ethenylidene and 2'-ethynyl cytidine, uridine and guanosine derivatives
EP0021231A3 (en) Process for the preparation of 5'-deoxy-5-fluoro uridine and intermediates in this process as well as their application
NO965483L (no) Dinukleosid-5',5'-pyrofosfater
DE3873470D1 (de) Behandlung von viruskrankheiten.
ES2164050T3 (es) Preparacion de compuestos de interleuquina-6.
SE7706425L (sv) Forfarande for framstellning av polyribonukleotider med aktivitet vid alstrandet av leukocyter och blodplettar
ES2085767T3 (es) Aumento de la actividad catalitica de las ribozimas por un oligonucleotido facilitador proximo.
ATE245652T1 (de) Macrolide mit antitumoraktivität
KR930018032A (ko) 복합효소를 이용한 l-오르니친의 제조방법
Eppstein et al. Bio—Organic Laboratory
SE8501666D0 (sv) Antracyklinderivat, forfaranden for framstellning derav samt farmaceutisk komposition innehallande dem

Legal Events

Date Code Title Description
8364 No opposition during term of opposition
8339 Ceased/non-payment of the annual fee