DE69123979D1 - Abgeänderte ribozyme - Google Patents

Abgeänderte ribozyme

Info

Publication number
DE69123979D1
DE69123979D1 DE69123979T DE69123979T DE69123979D1 DE 69123979 D1 DE69123979 D1 DE 69123979D1 DE 69123979 T DE69123979 T DE 69123979T DE 69123979 T DE69123979 T DE 69123979T DE 69123979 D1 DE69123979 D1 DE 69123979D1
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
DE69123979T2 (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 true DE69123979D1 (de) 1997-02-13
DE69123979T2 DE69123979T2 (de) 1997-04-30

Family

ID=8165527

Family Applications (2)

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

Family Applications After (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) DE69123979T2 (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
US5955589A (en) * 1991-12-24 1999-09-21 Isis Pharmaceuticals Inc. Gapped 2' modified oligonucleotides
US6399754B1 (en) 1991-12-24 2002-06-04 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides
US5670633A (en) * 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
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
US5623065A (en) * 1990-08-13 1997-04-22 Isis Pharmaceuticals, Inc. Gapped 2' modified oligonucleotides
US6005087A (en) 1995-06-06 1999-12-21 Isis Pharmaceuticals, Inc. 2'-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
PL169576B1 (pl) * 1990-10-12 1996-08-30 Max Planck Gesellschaft Sposób wytwarzania czasteczki RNA o aktywnosci katalitycznej PL PL
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
US5856455A (en) * 1991-12-24 1999-01-05 Isis Pharmaceuticals, Inc. Gapped 2'-modified oligonucleotides
US6277603B1 (en) 1991-12-24 2001-08-21 Isis Pharmaceuticals, Inc. PNA-DNA-PNA chimeric macromolecules
EP0618925B2 (de) * 1991-12-24 2012-04-18 Isis Pharmaceuticals, Inc. Antisense oligonukleotide
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
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
US5977343A (en) 1992-05-14 1999-11-02 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
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)
US20040054156A1 (en) * 1992-05-14 2004-03-18 Kenneth Draper Method and reagent for inhibiting hepatitis B viral replication
US20030125270A1 (en) * 2000-12-18 2003-07-03 Lawrence Blatt Enzymatic nucleic acid treatment of diseases or conditions related to hepatitis C virus infection
US5686599A (en) * 1992-05-14 1997-11-11 Ribozyme Pharmaceuticals, Inc. Synthesis, deprotection, analysis and purification of RNA and ribozymes
TW244371B (de) * 1992-07-23 1995-04-01 Tri Clover Inc
US6346614B1 (en) 1992-07-23 2002-02-12 Hybridon, Inc. Hybrid oligonucleotide phosphorothioates
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
IL107480A0 (en) * 1992-11-03 1994-02-27 Gene Shears Pty Ltd Tnf-x ribozymes and degradation resistant mrna derivatives linked to tnf-x ribozymes
US5864028A (en) * 1992-11-03 1999-01-26 Gene Shears Pty. Limited Degradation resistant mRNA derivatives linked to TNF-α ribozymes
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
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
US5811300A (en) * 1992-12-07 1998-09-22 Ribozyme Pharmaceuticals, Inc. TNF-α ribozymes
US5817635A (en) * 1993-08-09 1998-10-06 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
EP1253199A1 (de) 1993-09-02 2002-10-30 Ribozyme Pharmaceuticals, Inc. Enzymatische Nukleinsaüre die abasische Gruppen enthalten
AU7623194A (en) * 1993-09-03 1995-03-22 Vpi Holdings Ltd. Oligonucleotides with rna cleavage activity
US5861288A (en) * 1993-10-18 1999-01-19 Ribozyme Pharmaceuticals, Inc. Catalytic DNA
EP0725788B1 (de) * 1993-10-27 1998-12-16 Ribozyme Pharmaceuticals, Inc. 2'-amido-und 2'-peptido-modifizierte oligonukleotide
AU687479B2 (en) 1993-11-08 1998-02-26 Ribozyme Pharmaceuticals, Inc. Base-modified enzymatic nucleic acid
AU730347B2 (en) * 1993-11-08 2001-03-08 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
CA2183992A1 (en) * 1994-02-23 1995-08-31 Dan T. Stinchcomb Method and reagent for inhibiting the expression of disease related genes
EP1260586A3 (de) * 1994-02-23 2004-04-28 Ribozyme Pharmaceuticals, Inc. Verfahren und Verbindung zur Verringerung des Expression von Genen, die in Verbindung mit Krankheiten stehen
US5902880A (en) * 1994-08-19 1999-05-11 Ribozyme Pharmaceuticals, Inc. RNA polymerase III-based expression of therapeutic RNAs
US5693532A (en) * 1994-11-04 1997-12-02 Ribozyme Pharmaceuticals, Inc. Respiratory syncytial virus ribozymes
US5639647A (en) * 1994-03-29 1997-06-17 Ribozyme Pharmaceuticals, Inc. 2'-deoxy-2'alkylnucleotide containing nucleic acid
US5631359A (en) * 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
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
US5672501A (en) * 1994-12-23 1997-09-30 Ribozyme Pharmaceuticals, Inc. Base-modified enzymatic nucleic acid
US5716824A (en) 1995-04-20 1998-02-10 Ribozyme Pharmaceuticals, Inc. 2'-O-alkylthioalkyl and 2-C-alkylthioalkyl-containing enzymatic nucleic acids (ribozymes)
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
US5804560A (en) * 1995-01-06 1998-09-08 Sibia Neurosciences, Inc. Peptide and peptide analog protease inhibitors
US6017887A (en) * 1995-01-06 2000-01-25 Sibia Neurosciences, Inc. Peptide, peptide analog and amino acid 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
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
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
US20040220128A1 (en) * 1995-10-26 2004-11-04 Sirna Therapeutics, Inc. Nucleic acid based modulation of female reproductive diseases and conditions
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)
CA2237528A1 (en) * 1995-11-14 1997-05-22 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
DK0808898T3 (da) * 1996-05-24 2004-09-27 Aventis Pharma Gmbh Reagens og fremgangsmåde til inhibering af N-ras-ekspression
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US5898031A (en) * 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
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
US5807743A (en) * 1996-12-03 1998-09-15 Ribozyme Pharmaceuticals, Inc. Interleukin-2 receptor gamma-chain ribozymes
DE69738768D1 (de) * 1996-12-13 2008-07-24 Novartis Vaccines & Diagnostic Analyse und separation von pdgf-proteinen
JP2002514913A (ja) * 1996-12-19 2002-05-21 エール ユニバーシティ 生反応性アロステリックポリヌクレオチド
US6248878B1 (en) 1996-12-24 2001-06-19 Ribozyme Pharmaceuticals, Inc. Nucleoside analogs
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
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
US6159951A (en) * 1997-02-13 2000-12-12 Ribozyme Pharmaceuticals Inc. 2'-O-amino-containing nucleoside analogs and polynucleotides
KR20000075748A (ko) 1997-02-26 2000-12-26 마빈 씨. 구트리 알츠하이머 병의 치료 또는 예방에 유효한 약물을 선별하기 위한 유전자 변형동물 및 세포계
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
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
US6617438B1 (en) 1997-11-05 2003-09-09 Sirna Therapeutics, Inc. Oligoribonucleotides with enzymatic activity
US20020147143A1 (en) 1998-03-18 2002-10-10 Corixa Corporation Compositions and methods for the therapy and diagnosis of lung cancer
AU3751299A (en) 1998-04-20 1999-11-08 Ribozyme Pharmaceuticals, Inc. Nucleic acid molecules with novel chemical compositions capable of modulating gene expression
CA2230203A1 (en) 1998-04-29 1999-10-29 Universite De Sherbrooke Delta ribozyme for rna cleavage
AU6021799A (en) * 1998-08-26 2000-04-03 South Alabama Medical Science Foundation T-type calcium channel
US20030125269A1 (en) * 1998-08-26 2003-07-03 Ming Li T-type calcium channel
US6175004B1 (en) * 1998-09-01 2001-01-16 Isis Pharmaceuticals, Inc. Process for the synthesis of oligonucleotides incorporating 2-aminoadenosine
US6995259B1 (en) 1998-10-23 2006-02-07 Sirna Therapeutics, Inc. Method for the chemical synthesis of oligonucleotides
IL126732A0 (en) * 1998-10-23 1999-08-17 Intelligene Ltd Pro drug
EP1180114B1 (de) 1999-04-27 2011-09-21 The Trustees of The University of Pennsylvania Verwendung von Resistin spezifischen Antikörpern in der Behandlung von Diabetes
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
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)
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
US20050233329A1 (en) * 2002-02-20 2005-10-20 Sirna Therapeutics, Inc. Inhibition of gene expression using duplex forming oligonucleotides
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
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)
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)
AU3811101A (en) * 2000-02-11 2001-08-20 Lawrence Blatt Method and reagent for the modulation and diagnosis of cd20 and nogo gene expression
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
US7833992B2 (en) * 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
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
US20040009510A1 (en) * 2000-03-06 2004-01-15 Scott Seiwert Allosteric nucleic acid sensor molecules
US20030065155A1 (en) * 2000-03-06 2003-04-03 Nassim Usman Nucleic acid sensor molecules
CA2404890C (en) 2000-03-30 2013-11-19 Whitehead Institute For Biomedical Research Rna sequence-specific mediators of rna interference
ATE396265T1 (de) 2000-06-28 2008-06-15 Corixa Corp Zusammensetzungen und verfahren für therapie und diagnose von lungenkrebs
US20030190635A1 (en) * 2002-02-20 2003-10-09 Mcswiggen James A. RNA interference mediated treatment of Alzheimer's disease using short interfering RNA
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)
US20080032942A1 (en) * 2000-08-30 2008-02-07 Mcswiggen James RNA interference mediated treatment of Alzheimer's disease using short interfering nucleic acid (siNA)
BRPI0115814B8 (pt) 2000-12-01 2021-05-25 Europaeisches Laboratorium Fuer Molekularbiologie Embl moléculas de rna de filamento duplo, seu método de preparação e composição farmacêutica compreendendo as mesmas
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.
JP2004532022A (ja) * 2001-03-26 2004-10-21 サーナ・セラピューティクス・インコーポレイテッド B型肝炎ウイルスおよびc型肝炎ウイルスの複製のオリゴヌクレオチド媒介性阻害
AU2002305123B2 (en) 2001-03-30 2006-10-05 Philadelphia Health And Education Corporation Immunomodulation and effect on cell processes relating to serotonin family receptors
WO2002089747A2 (en) 2001-05-09 2002-11-14 Corixa Corporation Compositions and methods for the therapy and diagnosis of prostate cancer
US20080161256A1 (en) * 2001-05-18 2008-07-03 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acid (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)
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)
US20050282188A1 (en) * 2001-05-18 2005-12-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of 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
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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
US20050119211A1 (en) * 2001-05-18 2005-06-02 Sirna Therapeutics, Inc. RNA mediated inhibition connexin gene expression using short interfering nucleic acid (siNA)
WO2002097114A2 (en) * 2001-05-29 2002-12-05 Sirna Therapeutics, Inc. Nucleic acid treatment of diseases or conditions related to levels of ras, her2 and hiv
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)
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)
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)
US20070270579A1 (en) * 2001-05-18 2007-11-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of 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)
US20070042983A1 (en) * 2001-05-18 2007-02-22 Sirna Therapeutics, Inc. RNA interference mediated inhibition of 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)
US20050137155A1 (en) * 2001-05-18 2005-06-23 Sirna Therapeutics, Inc. RNA interference mediated treatment of Parkinson disease 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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
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)
US20030175950A1 (en) * 2001-05-29 2003-09-18 Mcswiggen James A. RNA interference mediated inhibition of HIV gene expression using short interfering RNA
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)
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)
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)
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
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)
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)
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)
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)
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)
US20040006035A1 (en) * 2001-05-29 2004-01-08 Dennis Macejak Nucleic acid mediated disruption of HIV fusogenic peptide interactions
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)
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)
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)
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
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)
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)
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)
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)
EP3231445A1 (de) 2001-05-18 2017-10-18 Sirna Therapeutics, Inc. Konjugate und zusammensetzungen zur zellulären verabreichung
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)
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
WO2003008628A2 (en) * 2001-07-20 2003-01-30 Ribozyme Pharmacuticals, Inc. Enzymatic nucleic acid peptide conjugates
AU2002365084A1 (en) * 2001-10-19 2003-07-24 Sirna Therapeutics, Inc Method and reagent for the detection of proteins and peptides
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)
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)
US20030219775A1 (en) * 2001-12-14 2003-11-27 Ward David C. Nucleic acid diagnostic reagents and methods for detecting nucleic acids, polynucleotides and oligonucleotides
ES2405790T3 (es) 2001-12-17 2013-06-03 Corixa Corporation Composiciones y métodos para la terapia y el diagnóstico de enfermedad inflamatoria del intestino
US20050042632A1 (en) * 2002-02-13 2005-02-24 Sirna Therapeutics, Inc. Antibodies having specificity for nucleic acids
US7071311B2 (en) * 2002-02-13 2006-07-04 Sirna Therapeutics, Inc. Antibodies having specificity for 2′-C-allyl 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)
WO2003106476A1 (en) * 2002-02-20 2003-12-24 Sirna Therapeutics, Inc Nucleic acid mediated inhibition of enterococcus infection and cytolysin toxin activity
US20050222064A1 (en) * 2002-02-20 2005-10-06 Sirna Therapeutics, Inc. Polycationic compositions for cellular delivery of polynucleotides
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)
AU2003207708A1 (en) 2002-02-20 2003-09-09 Sirna Therapeutics, Inc. Rna interference mediated inhibition of map kinase genes
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)
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)
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
CA2492463A1 (en) * 2002-07-25 2004-02-05 Archemix Corporation Regulated aptamer therapeutics
SI3222724T1 (sl) 2002-08-05 2019-03-29 Silence Therapeutics Gmbh Nadaljnje nove oblike molekul interferenčne RNA
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
US20040137471A1 (en) * 2002-09-18 2004-07-15 Timothy Vickers Efficient reduction of target RNA's by single-and double-stranded oligomeric compounds
WO2004029212A2 (en) 2002-09-25 2004-04-08 University Of Massachusetts In vivo gene silencing by chemically modified and stable sirna
US8604183B2 (en) 2002-11-05 2013-12-10 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
US20050124565A1 (en) * 2002-11-21 2005-06-09 Diener John L. Stabilized aptamers to platelet derived growth factor and their use as oncology therapeutics
US10100316B2 (en) * 2002-11-21 2018-10-16 Archemix Llc Aptamers comprising CPG motifs
US8853376B2 (en) 2002-11-21 2014-10-07 Archemix Llc Stabilized aptamers to platelet derived growth factor and their use as oncology therapeutics
US8039443B2 (en) 2002-11-21 2011-10-18 Archemix Corporation Stabilized aptamers to platelet derived growth factor and their use as oncology therapeutics
US20040180360A1 (en) * 2002-11-21 2004-09-16 Charles Wilson Multivalent aptamer therapeutics with improved pharmacodynamic properties and methods of making and using the same
WO2004050899A2 (en) * 2002-12-03 2004-06-17 Archemix Corporation Method for in vitro selection of 2’-substituted nucleic acids
US20050037394A1 (en) * 2002-12-03 2005-02-17 Keefe Anthony D. Method for in vitro selection of 2'-substituted nucleic acids
US20040209353A1 (en) * 2002-12-12 2004-10-21 Chiron Corporation Biological sample storage device and method for biological sample contamination testing
US20040231231A1 (en) * 2002-12-20 2004-11-25 Cataldo Dominic A. Use of colloidal clays for sustained release of active ingredients
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
WO2004060323A2 (en) * 2003-01-02 2004-07-22 The Research Foundation Of State University Of Newyork Novel inhibitors of poxvirus replication
EP1613269B1 (de) 2003-04-04 2015-02-25 Incyte Corporation Zusammensetzungen, verfahren und kits in zusammenhang mit her-2 spaltung
AU2004231740A1 (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
CA2523260A1 (en) 2003-04-21 2004-11-04 Archemix Corporation Stabilized aptamers to platelet derived growth factor and their use as oncology therapeutics
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
US20060019914A1 (en) * 2004-02-11 2006-01-26 University Of Tennessee Research Foundation Inhibition of tumor growth and invasion by anti-matrix metalloproteinase DNAzymes
EP1727567B1 (de) 2004-02-12 2013-07-03 Archemix LLC Zur Behandlung von Erkrankungen in Verbindung mit dem Komplementsystem geeignete Aptamer-Therapeutika
US7803931B2 (en) 2004-02-12 2010-09-28 Archemix Corp. Aptamer therapeutics useful in the treatment of complement-related disorders
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
AU2005220910A1 (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
CA2557532A1 (en) * 2004-04-23 2005-11-10 Angela M. Christiano 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
WO2006112869A2 (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
EP1789096A4 (de) 2004-09-07 2009-07-08 Archemix Corp Aptamere für den von-willebrand-faktor und ihre verwendung als therapeutika für thrombotische erkrankungen
KR20070101226A (ko) 2004-09-07 2007-10-16 아케믹스 코포레이션 앱타머의 약화학
US7566701B2 (en) 2004-09-07 2009-07-28 Archemix Corp. Aptamers to von Willebrand Factor and their use as thrombotic disease therapeutics
US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
EP2436391A3 (de) 2004-11-02 2012-07-04 Archemix LLC Stabilisierte Aptamere für einen aus Plättchen gewonnenen Wachstumsfaktor und ihre Verwendung als Krebstherapiemittel
ES2401285T3 (es) 2004-12-16 2013-04-18 The Regents Of The University Of California Fármacos con el pulmón como diana
CA2600418A1 (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
ATE522626T1 (de) 2005-06-28 2011-09-15 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
KR20080025181A (ko) 2005-06-30 2008-03-19 아케믹스 코포레이션 완전 2'-변형된 핵산 전사체를 생성하기 위한 물질 및 방법
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
EP1981902B1 (de) 2006-01-27 2015-07-29 Biogen MA Inc. Nogo-rezeptorantagonisten
US7922000B2 (en) * 2006-02-15 2011-04-12 Miraial Co., Ltd. Thin plate container with a stack of removable loading trays
RU2477137C2 (ru) 2006-03-08 2013-03-10 АРКЕМИКС Эл Эл Си Связывающие комплемент аптамеры и средства против с5, пригодные для лечения глазных нарушений
WO2007115168A2 (en) 2006-03-31 2007-10-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of eg5 gene
EP2010226B1 (de) 2006-04-07 2014-01-15 The Research Foundation of State University of New York Transcobalaminrezeptor-Polypeptide, -Nukleinsäuren und -Modulatoren sowie entsprechende Verfahren zur Verwendung für die Modulation von Zellwachstum und die Behandlung von Krebs und Cobalaminmangel
EP2016060A4 (de) * 2006-05-05 2010-08-04 Wallac Oy Auf azacycloalkanen basierende reagentien zum markieren von biomolekülen und davon abgeleitete konjugate
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
CN101511181B (zh) 2006-07-11 2013-08-21 新泽西医科和牙科大学 蛋白、编码该蛋白的核酸和相关的应用方法
US9505821B2 (en) 2006-10-03 2016-11-29 Rutgers, The State University Of New Jersey ATAP peptides, nucleic acids encoding the same and associated methods of use
US20100166743A1 (en) 2006-10-06 2010-07-01 University Of Utah Research Foundation Method of detecting ocular diseases and pathologic conditions and treatment of same
EP2061901A2 (de) 2006-10-31 2009-05-27 Noxxon Pharma AG Verfahren zum nachweis eines einzel- oder doppelsträngigen nukleinsäuremoleküls
WO2008094370A2 (en) 2006-12-22 2008-08-07 University Of Utah Research Foundation Method of detecting ocular diseases and pathologic conditions and treatment of same
EP2146691A2 (de) * 2007-04-17 2010-01-27 Baxter International Inc. Nukleinsäure-mikroteilchen zur abgabe in den lungen
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
WO2008147526A1 (en) 2007-05-23 2008-12-04 The Trustees Of The University Of Pennsylvania Targeted carriers for intracellular drug delivery
WO2009033027A2 (en) 2007-09-05 2009-03-12 Medtronic, Inc. Suppression of scn9a gene expression and/or function for the treatment of pain
US20090094716A1 (en) * 2007-09-24 2009-04-09 Performance Plants, Inc. Plants having increased biomass
JP2011505144A (ja) 2007-11-30 2011-02-24 ベイラー カレッジ オブ メディシン 樹状細胞ワクチン組成物およびその使用
CA2708153C (en) 2007-12-04 2017-09-26 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
EP3100718B1 (de) 2008-01-02 2019-11-27 Arbutus Biopharma Corporation Verbesserte zusammensetzungen und verfahren zur freisetzung von nukleinsäuren
BRPI0911332A2 (pt) 2008-04-04 2019-09-24 Calando Pharmaceuticals Inc composições e uso de inibidores de epas1
PL2279254T3 (pl) 2008-04-15 2017-11-30 Protiva Biotherapeutics Inc. Nowe preparaty lipidowe do dostarczania kwasów nukleinowych
WO2009137095A2 (en) 2008-05-08 2009-11-12 The Johns Hopkins University Compositions and methods for modulating an immune response
US20110212021A1 (en) 2008-05-30 2011-09-01 Slack Frank J Targeted oligonucleotide compositions for modifying gene expression
AU2009274512A1 (en) 2008-07-25 2010-01-28 The Regents Of The University Of Colorado Clip inhibitors and methods of modulating immune function
AU2009290102A1 (en) * 2008-09-05 2010-03-11 The Royal Institution For The Advancement Of Learning/Mcgill University RNA monomers containing O-acetal levulinyl ester groups and their use in RNA microarrays
EP2341924A4 (de) 2008-10-02 2013-01-23 David Gladstone Inst Verfahren zur behandlung von hepatitis-c-virusinfektionen
US9139554B2 (en) 2008-10-09 2015-09-22 Tekmira Pharmaceuticals Corporation Amino lipids and methods for the delivery of nucleic acids
CN104382853A (zh) 2008-10-16 2015-03-04 玛瑞纳生物技术有限公司 基因沉默治疗剂的脂质体有效递送方法和组合物
WO2010051105A1 (en) 2008-10-29 2010-05-06 China Synthetic Rubber Corporation Methods and agents for the diagnosis and treatment of hepatocellular carcinoma
EP4241767A3 (de) 2008-11-10 2023-11-01 Arbutus Biopharma Corporation Neuartige lipide und zusammensetzungen zur verabreichung von therapeutika
EP3243504A1 (de) 2009-01-29 2017-11-15 Arbutus Biopharma Corporation Verbesserte lipidformulierung
US20100215660A1 (en) 2009-02-23 2010-08-26 Sarwar Hashmi Kruppel-like factors and fat regulation
JP6010458B2 (ja) 2009-04-03 2016-10-19 ダイセルナ ファーマシューティカルズ, インコーポレイテッドDicerna Pharmaceuticals, Inc. 非対称二本鎖rnaによる特異的阻害のための、方法および組成物
EP2756845B1 (de) 2009-04-03 2017-03-15 Dicerna Pharmaceuticals, Inc. Verfahren und Zusammensetzungen für spezifische KRAS-Hemmung mit asymmetrischer Doppelstrang-RNA
US20120149756A1 (en) 2009-04-10 2012-06-14 Associatin Institut de Myologie Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease
US9724404B2 (en) 2009-04-13 2017-08-08 INSERM (Institut National de la Santé et de la Recherche Médicale) HPV particles and uses thereof
NZ621981A (en) 2009-05-05 2015-09-25 Tekmira Pharmaceuticals Corp Lipid compositions
CA3045126A1 (en) 2009-05-05 2010-11-11 Arbutus Biopharma Corporation Methods of delivering oligonucleotides to immune cells
EP2430168B1 (de) 2009-05-16 2016-12-21 Kunyuan Cui Zusammensetzung mit kationischen amphiphilen und colipiden zur abgabe von heilmittelmolekülen
HUE056773T2 (hu) 2009-06-10 2022-03-28 Arbutus Biopharma Corp Továbbfejlesztett lipid készítmény
EP2449114B9 (de) 2009-07-01 2017-04-19 Protiva Biotherapeutics Inc. Neue lipidformulierungen zur abgabe von therapiemitteln in feste tumore
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
CA2783372C (en) 2009-12-07 2019-07-16 Muthiah Manoharan Compositions for nucleic acid delivery
ES2749426T3 (es) 2009-12-18 2020-03-20 Univ British Columbia Métodos y composiciones para administración de ácidos nucleicos
CN102770767A (zh) 2010-02-10 2012-11-07 诺瓦提斯公司 用于肌肉生长的方法和组合物
ES2647390T3 (es) 2010-02-11 2017-12-21 Nanostring Technologies, Inc Composiciones y métodos para la detección preferencial de ARN pequeños mediante hibridación con puente y ligación
US9080171B2 (en) 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
WO2011119842A1 (en) 2010-03-25 2011-09-29 The J. David Gladstone Institutes Compositions and methods for treating neurological disorders
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
US9816108B2 (en) 2010-04-23 2017-11-14 University Of Florida Research Foundation, Inc. rAAV-guanylate cyclase compositions and methods for treating lebers congenital amaurosis-1 (LCA1)
US20130190383A1 (en) 2010-04-26 2013-07-25 Marina Biotech, Inc. Nucleic acid compounds with conformationally restricted monomers and uses thereof
WO2011139842A2 (en) 2010-04-28 2011-11-10 Marina Biotech, Inc. Nucleic acid compounds for inhibiting fgfr3 gene expression and uses thereof
WO2012006243A2 (en) 2010-07-06 2012-01-12 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of beta-catenin by double-stranded rna
WO2012006506A1 (en) 2010-07-09 2012-01-12 Massachusetts Institute Of Technology Metabolic gene, enzyme, and flux targets for cancer therapy
US20130202652A1 (en) 2010-07-30 2013-08-08 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
US20130323269A1 (en) 2010-07-30 2013-12-05 Muthiah Manoharan Methods and compositions for delivery of active agents
WO2012027206A1 (en) 2010-08-24 2012-03-01 Merck Sharp & Dohme Corp. SINGLE-STRANDED RNAi AGENTS CONTAINING AN INTERNAL, NON-NUCLEIC ACID SPACER
SG188666A1 (en) 2010-09-30 2013-05-31 Agency Science Tech & Res Methods and reagents for detection and treatment of esophageal metaplasia
US9260471B2 (en) 2010-10-29 2016-02-16 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using short interfering nucleic acids (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)
WO2012099755A1 (en) 2011-01-11 2012-07-26 Alnylam Pharmaceuticals, Inc. Pegylated lipids and their use for drug delivery
CA2824235A1 (en) 2011-01-11 2012-07-19 Fate Therapeutics, Inc. Novel wnt compositions and therapeutic uses of such compositions
SG11201401314PA (en) 2011-09-07 2014-09-26 Marina Biotech Inc Synthesis and uses of nucleic acid compounds with conformationally restricted monomers
WO2013049328A1 (en) 2011-09-27 2013-04-04 Alnylam Pharmaceuticals, Inc. Di-aliphatic substituted pegylated lipids
WO2013055789A1 (en) 2011-10-14 2013-04-18 Accugenomics, Inc. Nucleic acid amplification and use thereof
KR102102862B1 (ko) 2011-10-14 2020-04-22 제넨테크, 인크. 항-HtrA1 항체 및 사용 방법
DK2790736T3 (en) 2011-12-12 2018-05-07 Oncoimmunin Inc In vivo delivery of oligonucleotides
US9562072B2 (en) 2011-12-27 2017-02-07 The J. David Gladstone Institutes Compositions and methods for regulating glucose metabolism
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
BR112014020824B1 (pt) 2012-02-24 2022-10-04 Protiva Biotherapeutics Inc Lipídeo, partícula de lipídeo e composição farmacêutica
CA2874972C (en) 2012-05-25 2023-06-20 Accugenomics, Inc. Nucleic acid amplification and use thereof
US11142762B2 (en) 2012-07-05 2021-10-12 The Trustees Of The University Of Pennsylvania U1 snRNP regulates gene expression and modulates oncogenicity
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
ES2912033T3 (es) 2012-10-23 2022-05-24 Caris Science Inc Aptámeros y usos de los mismos
US10942184B2 (en) 2012-10-23 2021-03-09 Caris Science, Inc. Aptamers and uses thereof
US9939443B2 (en) 2012-12-19 2018-04-10 Caris Life Sciences Switzerland Holdings Gmbh Compositions and methods for aptamer screening
KR102092435B1 (ko) 2013-03-08 2020-03-24 예일 유니버시티 피부색소침착 감소용 조성물 및 방법
CN107056838A (zh) 2013-03-15 2017-08-18 加利福尼亚大学董事会 无环核苷膦酸二酯
JP2016528887A (ja) 2013-07-03 2016-09-23 ダイセルナ ファーマシューティカルズ, インコーポレイテッドDicerna Pharmaceuticals, Inc. 二本鎖RNAによるα−1アンチトリプシンの特異的阻害のための方法及び組成物
JP6702862B2 (ja) 2013-07-11 2020-06-03 アルニラム ファーマスーティカルズ インコーポレイテッドAlnylam Pharmaceuticals, Inc. オリゴヌクレオチド−リガンドコンジュゲートおよびそれらの調製方法
US20150044192A1 (en) 2013-08-09 2015-02-12 President And Fellows Of Harvard College Methods for identifying a target site of a cas9 nuclease
JP2016533752A (ja) 2013-08-28 2016-11-04 カリス ライフ サイエンシズ スウィッツァーランド ホー オリゴヌクレオチドプローブおよびその使用
JP6444417B2 (ja) 2013-09-18 2018-12-26 オーラ バイオサイエンシーズ, インコーポレイテッド 腫瘍を診断および処置するためのウイルス様粒子結合体
EP3049085B9 (de) 2013-09-26 2021-08-18 Beth Israel Deaconess Medical Center, Inc. Sgk1 inhibitoren bei der behandlung von long-qt-syndrom
US9365610B2 (en) 2013-11-18 2016-06-14 Arcturus Therapeutics, Inc. Asymmetric ionizable cationic lipid for RNA delivery
WO2015074085A1 (en) 2013-11-18 2015-05-21 Arcturus Therapeutics, Inc. 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
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
US10934550B2 (en) 2013-12-02 2021-03-02 Phio Pharmaceuticals Corp. Immunotherapy of cancer
JP7017309B2 (ja) 2013-12-20 2022-02-08 アセチロン ファーマシューティカルズ インコーポレイテッド 多発性骨髄腫におけるヒストン脱アセチル化酵素6(hdac6)バイオマーカー
US9701966B2 (en) 2013-12-27 2017-07-11 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of glycolate oxidase (HAO1) by double-stranded RNA
US9458464B2 (en) 2014-06-23 2016-10-04 The Johns Hopkins University Treatment of neuropathic pain
ES2903029T3 (es) 2014-07-03 2022-03-30 Univ Yale La inhibición de DICKKOPF2 (DKK2) suprime la formación de tumores
WO2016022363A2 (en) 2014-07-30 2016-02-11 President And Fellows Of Harvard College Cas9 proteins including ligand-dependent inteins
HRP20220651T1 (hr) 2014-09-15 2022-08-19 The Regents Of The University Of California Nukleotidni analozi
CA2964168A1 (en) 2014-10-10 2016-04-14 Dicerna Pharmaceuticals, Inc. Therapeutic inhibition of lactate dehydrogenase and agents therefor
EP3206751A4 (de) 2014-10-14 2018-06-13 The J. David Gladstone Institutes Zusammensetzungen und verfahren zur reaktivierung von latentem immundefizienzvirus
WO2016081029A1 (en) 2014-11-18 2016-05-26 Arcturus Therapeutics, Inc. Ionizable cationic lipid for rna delivery
US20170355755A1 (en) 2014-11-21 2017-12-14 Yale University Compositions and methods for modulating salm5 and hvem
DK3569711T3 (da) 2014-12-15 2021-02-22 Dicerna Pharmaceuticals Inc Ligandmodificerede dobbeltstrengede nukleinsyrer
JP2017538427A (ja) 2014-12-18 2017-12-28 インテグレイテッド ディーエヌエイ テクノロジーズ インコーポレイテッド Crispr系組成物及び使用方法
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
WO2016145128A1 (en) 2015-03-09 2016-09-15 Caris Science, Inc. Oligonucleotide probes and uses thereof
CA2991045A1 (en) 2015-06-29 2017-01-05 Caris Science, Inc. Therapeutic oligonucleotides binding c1q
WO2017019918A1 (en) 2015-07-28 2017-02-02 Caris Science, Inc. Targeted oligonucleotides
US11273167B2 (en) 2015-08-03 2022-03-15 The Regents Of The University Of California Compositions and methods for modulating ABHD2 activity
KR20180095511A (ko) 2015-10-30 2018-08-27 더 유나이티드 스테이츠 오브 어메리카, 애즈 리프리젠티드 바이 더 세크러테리, 디파트먼트 오브 헬쓰 앤드 휴먼 서비씨즈 표적화된 암 요법
IL295097A (en) 2015-10-30 2022-09-01 Genentech Inc Anti-htra1 antibodies and methods of using them
CN109715802A (zh) 2016-03-18 2019-05-03 卡里斯科学公司 寡核苷酸探针及其用途
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
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
MA45328A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
MA45469A (fr) 2016-04-01 2019-02-06 Avidity Biosciences Llc Acides nucléiques de bêta-caténine et leurs utilisations
EP3448369A4 (de) 2016-04-29 2020-01-01 The Regents of The University of Colorado, A Body Corporate Verbindungen und zusammensetzungen zur behandlung des stoffwechselsyndroms und verfahren damit
WO2017205686A1 (en) 2016-05-25 2017-11-30 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
EP3500696A4 (de) 2016-08-16 2020-04-08 Bluebird Bio, Inc. Il-10-rezeptor-alpha-homing-endonuklease-varianten, zusammensetzungen und verfahren zur verwendung
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
JP2019533996A (ja) 2016-10-07 2019-11-28 インテグレイテツド・デイー・エヌ・エイ・テクノロジーズ・インコーポレイテツド S.ピオゲネスcas9変異遺伝子及びこれによってコードされるポリペプチド
DK3541833T3 (da) 2016-11-17 2024-04-02 2Seventy Bio Inc TGFß signalkonverter
WO2018098383A1 (en) 2016-11-22 2018-05-31 Integrated Dna Technologies, Inc. Crispr/cpf1 systems and methods
WO2018111947A1 (en) 2016-12-12 2018-06-21 Integrated Dna Technologies, Inc. Genome editing enhancement
US10526284B2 (en) 2016-12-21 2020-01-07 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
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
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
EP3583203B1 (de) 2017-02-15 2023-11-01 2seventy bio, Inc. Spenderreparaturtemplates für multiplex-genomeditierung
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
EP3658573A1 (de) 2017-07-28 2020-06-03 President and Fellows of Harvard College Verfahren und zusammensetzungen zur entwicklung von basiseditoren unter verwendung von phagenunterstützter kontinuierlicher entwicklung (pace)
US11319532B2 (en) 2017-08-30 2022-05-03 President And Fellows Of Harvard College High efficiency base editors comprising Gam
US11306312B2 (en) 2017-09-01 2022-04-19 Thomas Jefferson University Compositions and methods for MYC messenger RNA inhibitors
CA3077213A1 (en) 2017-10-04 2019-04-11 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
EP3697906A1 (de) 2017-10-16 2020-08-26 The Broad Institute, Inc. Verwendungen von adenosinbasiseditoren
CN111902148A (zh) 2017-12-06 2020-11-06 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
WO2019136459A1 (en) 2018-01-08 2019-07-11 Iovance Biotherapeutics, Inc. Processes for generating til products enriched for tumor antigen-specific t-cells
US11713446B2 (en) 2018-01-08 2023-08-01 Iovance Biotherapeutics, Inc. Processes for generating TIL products enriched for tumor antigen-specific T-cells
MX2020007046A (es) 2018-01-08 2020-09-07 Iovance Biotherapeutics Inc Procesos para generar productos de linfocitos infiltrantes de tumores (til) enriquecidos para celulas t especificas de antigenos tumorales.
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
EP3891274B1 (de) 2018-12-06 2023-10-18 Arcturus Therapeutics, Inc. Zusammensetzungen und verfahren zur behandlung von ornithintranscarbamylase-mangel
DE112020001342T5 (de) 2019-03-19 2022-01-13 President and Fellows of Harvard College Verfahren und Zusammensetzungen zum Editing von Nukleotidsequenzen
US20220204975A1 (en) 2019-04-12 2022-06-30 President And Fellows Of Harvard College System for genome editing
AU2020289464A1 (en) 2019-06-06 2022-01-20 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
PE20220968A1 (es) 2019-08-14 2022-06-10 Acuitas Therapeutics Inc Nanoparticulas lipidicas mejoradas para el suministro de acidos nucleicos
EP4121063A1 (de) 2020-03-19 2023-01-25 Avidity Biosciences, Inc. Zusammensetzungen und verfahren zur behandlung von fazioskapulohumeraler muskeldystrophie
CA3177180A1 (en) 2020-03-27 2021-09-30 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
AU2021374966A1 (en) 2020-11-09 2023-06-15 1E Therapeutics, Ltd. Catalytic sequence based methods of treating or preventing bacterial infections
WO2022130388A2 (en) 2020-12-18 2022-06-23 Yeda Research And Development Co. Ltd. Compositions for use in the treatment of chd2 haploinsufficiency and methods of identifying same
WO2022144882A2 (en) 2020-12-28 2022-07-07 1E Therapeutics, Ltd. P21 mrna target areas for silencing
IL304068A (en) 2020-12-28 2023-08-01 1E Therapeutics Ltd DNA enzymes against p21 messenger RNA
EP4288140A1 (de) 2021-02-05 2023-12-13 Iovance Biotherapeutics, Inc. Adjuvante therapie für krebs
EP4314016A1 (de) 2021-03-31 2024-02-07 Entrada Therapeutics, Inc. Zyklische zellen penetrierende peptide
EP4334437A1 (de) 2021-05-03 2024-03-13 Astellas Institute for Regenerative Medicine Verfahren zur erzeugung reifer hornhautendothelzellen
EP4337263A1 (de) 2021-05-10 2024-03-20 Entrada Therapeutics, Inc. Zusammensetzungen und verfahren zur modulation der interferon-regulatorischen faktor-5 (irf-5)-aktivität
KR20240012425A (ko) 2021-05-10 2024-01-29 엔트라다 테라퓨틱스, 인크. 세포내 치료제를 위한 조성물 및 방법
CA3217463A1 (en) 2021-05-10 2022-11-17 Ziqing QIAN Compositions and methods for modulating mrna splicing
EP4355875A1 (de) 2021-06-14 2024-04-24 2seventy bio, Inc. Verfahren zur aufreinigung einzelsträngiger rna
WO2022271955A1 (en) 2021-06-23 2022-12-29 Musc Foundation For Research Development Novel targeted shrna nanoparticles for cancer therapy
WO2022271818A1 (en) 2021-06-23 2022-12-29 Entrada Therapeutics, Inc. Antisense compounds and methods for targeting cug repeats
US20230138928A1 (en) 2021-08-05 2023-05-04 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
CA3233113A1 (en) 2021-09-22 2023-03-30 Sanegene Bio Usa Inc. 2'-alkyl or 3'-alkyl modified ribose derivatives for use in the in-vivo delivery of oligonucleotides
AU2022361238A1 (en) 2021-10-05 2024-04-11 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
US20230346819A1 (en) 2022-02-22 2023-11-02 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
EP0640688A1 (de) * 1987-12-15 1995-03-01 Gene Shears Pty. Limited Ribozyme
ES2090049T3 (es) * 1989-03-16 1996-10-16 Boehringer Ingelheim Int Unidades geneticas para la inhibicion de la funcion de arn.
CA2039718C (en) * 1989-08-31 2003-02-25 John J. Rossi Chimeric dna-rna catalytic sequences
US5149796A (en) * 1989-08-31 1992-09-22 City Of Hope Chimeric DNA-RNA catalytic sequences
WO1991019789A1 (en) * 1990-06-19 1991-12-26 Commonwealth Scientific And Industrial Research Organisation Endonucleases
PL169576B1 (pl) * 1990-10-12 1996-08-30 Max Planck Gesellschaft Sposób wytwarzania czasteczki RNA o aktywnosci katalitycznej PL PL
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

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EP0552178B1 (de) 1997-01-02
GR930300130T1 (en) 1993-12-30
CA2093664A1 (en) 1992-04-13
US5672695A (en) 1997-09-30
PL169576B1 (pl) 1996-08-30
CA2093664C (en) 2003-07-29
ES2061416T3 (es) 1997-03-01
WO1992007065A1 (en) 1992-04-30
JP2002101893A (ja) 2002-04-09
DE69123979T2 (de) 1997-04-30
EP0552178A1 (de) 1993-07-28
JPH06501842A (ja) 1994-03-03
ES2061416T1 (es) 1994-12-16
JP3257675B2 (ja) 2002-02-18
US6890908B1 (en) 2005-05-10
ATE147098T1 (de) 1997-01-15
US5698687A (en) 1997-12-16
DE552178T1 (de) 1994-02-03
AU8613991A (en) 1992-05-20
AU649074B2 (en) 1994-05-12

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