KR100211552B1 - 유전자 발현 억제용 화합물 및 방법 - Google Patents

유전자 발현 억제용 화합물 및 방법 Download PDF

Info

Publication number
KR100211552B1
KR100211552B1 KR1019930700317A KR930700317A KR100211552B1 KR 100211552 B1 KR100211552 B1 KR 100211552B1 KR 1019930700317 A KR1019930700317 A KR 1019930700317A KR 930700317 A KR930700317 A KR 930700317A KR 100211552 B1 KR100211552 B1 KR 100211552B1
Authority
KR
South Korea
Prior art keywords
independently
hydrogen
compound
alkyl
oxygen
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
KR1019930700317A
Other languages
English (en)
Korean (ko)
Other versions
KR930702372A (ko
Inventor
알렉산더루드빅 웨이스
프레데릭헤르만 하우셔
프라사드벤카타칼라 카투르베둘라
다니엘죠셉 델레키
폴프랜시스쥬니어 카바나프
패트리시아수잔 모스콰
프레드테리 오아키스
Original Assignee
디. 꼬쉬
사노피
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
Priority claimed from US07/562,180 external-priority patent/US5245022A/en
Application filed by 디. 꼬쉬, 사노피 filed Critical 디. 꼬쉬
Publication of KR930702372A publication Critical patent/KR930702372A/ko
Application granted granted Critical
Publication of KR100211552B1 publication Critical patent/KR100211552B1/ko
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Organic Low-Molecular-Weight Compounds And Preparation Thereof (AREA)
  • Golf Clubs (AREA)
  • Adhesives Or Adhesive Processes (AREA)
KR1019930700317A 1990-08-03 1991-08-02 유전자 발현 억제용 화합물 및 방법 Expired - Fee Related KR100211552B1 (ko)

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US07/562,180 US5245022A (en) 1990-08-03 1990-08-03 Exonuclease resistant terminally substituted oligonucleotides
US262,180 1990-08-03
US562,180 1990-08-03
US58245690A 1990-09-13 1990-09-13
US58228790A 1990-09-13 1990-09-13
US58245790A 1990-09-13 1990-09-13
US582,287 1990-09-13
US582,457 1990-09-13
US582,456 1990-09-13
US68278491A 1991-04-09 1991-04-09
US682,784 1991-04-09
PCT/US1991/005531 WO1992002534A2 (en) 1990-08-03 1991-08-02 Compounds and methods for inhibiting gene expression

Publications (2)

Publication Number Publication Date
KR930702372A KR930702372A (ko) 1993-09-08
KR100211552B1 true KR100211552B1 (ko) 1999-08-02

Family

ID=27541908

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1019930700317A Expired - Fee Related KR100211552B1 (ko) 1990-08-03 1991-08-02 유전자 발현 억제용 화합물 및 방법

Country Status (20)

Country Link
US (1) US5677439A (enExample)
EP (1) EP0541722B1 (enExample)
JP (1) JPH06502300A (enExample)
KR (1) KR100211552B1 (enExample)
AT (1) ATE131827T1 (enExample)
AU (2) AU667459B2 (enExample)
BR (1) BR9106729A (enExample)
CA (1) CA2088673A1 (enExample)
DE (1) DE69115702T2 (enExample)
DK (1) DK0541722T3 (enExample)
ES (1) ES2083593T3 (enExample)
FI (1) FI930455L (enExample)
GR (1) GR3018881T3 (enExample)
HU (2) HU217036B (enExample)
IL (3) IL99066A (enExample)
MY (1) MY107332A (enExample)
NZ (1) NZ239247A (enExample)
PT (1) PT98562B (enExample)
TW (1) TW222641B (enExample)
WO (1) WO1992002534A2 (enExample)

Families Citing this family (862)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5914396A (en) * 1990-01-11 1999-06-22 Isis Pharmaceuticals, Inc. 2'-O-modified nucleosides and phosphoramidites
US5623070A (en) * 1990-07-27 1997-04-22 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5677437A (en) * 1990-07-27 1997-10-14 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US6087482A (en) * 1990-07-27 2000-07-11 Isis Pharmaceuticals, Inc. Heteroatomic oligonucleoside linkages
US5792844A (en) * 1990-07-27 1998-08-11 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent nitrogen atoms
US5378825A (en) * 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5602240A (en) * 1990-07-27 1997-02-11 Ciba Geigy Ag. Backbone modified oligonucleotide analogs
US5618704A (en) * 1990-07-27 1997-04-08 Isis Pharmacueticals, Inc. Backbone-modified oligonucleotide analogs and preparation thereof through radical coupling
US5386023A (en) * 1990-07-27 1995-01-31 Isis Pharmaceuticals Backbone modified oligonucleotide analogs and preparation thereof through reductive coupling
US5610289A (en) * 1990-07-27 1997-03-11 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogues
US5783682A (en) 1990-07-27 1998-07-21 Isis Pharmaceuticals, Inc. Oligonucleotide mimics having nitrogen-containing linkages
PT98562B (pt) * 1990-08-03 1999-01-29 Sanofi Sa Processo para a preparacao de composicoes que compreendem sequencias de nucleo-sidos com cerca de 6 a cerca de 200 bases resistentes a nucleases
US5965722A (en) 1991-05-21 1999-10-12 Isis Pharmaceuticals, Inc. Antisense inhibition of ras gene with chimeric and alternating oligonucleotides
US6030954A (en) * 1991-09-05 2000-02-29 University Of Connecticut Targeted delivery of poly- or oligonucleotides to cells
US6335434B1 (en) 1998-06-16 2002-01-01 Isis Pharmaceuticals, Inc., Nucleosidic and non-nucleosidic folate conjugates
US8153602B1 (en) 1991-11-19 2012-04-10 Isis Pharmaceuticals, Inc. Composition and methods for the pulmonary delivery of nucleic acids
US5817781A (en) * 1992-06-01 1998-10-06 Gilead Sciences, Inc. Modified internucleoside linkages (II)
AU685080B2 (en) * 1993-01-07 1998-01-15 Thomas Jefferson University Antisense inhibition of C-MYC to modulate the proliferation of smooth muscle cells
GB9304618D0 (en) * 1993-03-06 1993-04-21 Ciba Geigy Ag Chemical compounds
WO1994022893A1 (en) * 1993-03-30 1994-10-13 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent nitrogen atoms
EP0701564A1 (en) * 1993-03-31 1996-03-20 Sanofi Bifunctional nucleosides, oligomers thereof, and methods of making and using the same
EP0691979A1 (en) * 1993-03-31 1996-01-17 Sanofi Novel 5'-substituted nucleosides and oligomers produced therefrom
DE69433036T2 (de) 1993-09-03 2004-05-27 Isis Pharmaceuticals, Inc., Carlsbad Aminoderivatisierte nukleoside und oligonukleoside
US6448373B1 (en) 1994-01-11 2002-09-10 Isis Pharmaceuticals, Inc. Phosphate linked oligomers formed of monomeric diols and processes for preparing same
US5886177A (en) * 1994-01-11 1999-03-23 Isis Pharmaceuticals, Inc. Phosphate linked oligomers
DE69527942T2 (de) * 1994-01-26 2003-03-27 Novartis Ag, Basel Modifizierte oligonukleotide
EP1260586A3 (en) * 1994-02-23 2004-04-28 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting the expression of disease related genes
FR2733500B1 (fr) * 1995-04-28 1997-07-25 Centre Nat Rech Scient Nouveaux antisens diriges contre ras, preparation et utilisations
US6420549B1 (en) 1995-06-06 2002-07-16 Isis Pharmaceuticals, Inc. Oligonucleotide analogs having modified dimers
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
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
US20040161844A1 (en) * 1996-06-06 2004-08-19 Baker Brenda F. Sugar and backbone-surrogate-containing oligomeric compounds and compositions for use in gene modulation
US9096636B2 (en) 1996-06-06 2015-08-04 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
US7812149B2 (en) 1996-06-06 2010-10-12 Isis Pharmaceuticals, Inc. 2′-Fluoro substituted oligomeric compounds and compositions for use in gene modulations
US6716625B1 (en) 1997-04-16 2004-04-06 Claude Selitrennikoff Histidine kinases of Aspergillus and other fungal species, related compositions, and methods of use
AU731909B2 (en) 1997-07-01 2001-04-05 Isis Pharmaceuticals, Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
US20040186071A1 (en) 1998-04-13 2004-09-23 Bennett C. Frank Antisense modulation of CD40 expression
US7321828B2 (en) 1998-04-13 2008-01-22 Isis Pharmaceuticals, Inc. System of components for preparing oligonucleotides
JP2002515514A (ja) 1998-05-21 2002-05-28 アイシス・ファーマシューティカルス・インコーポレーテッド オリゴヌクレオチドの局所送逹のための組成物及び方法
AU745880B2 (en) * 1998-05-21 2002-04-11 Isis Pharmaceuticals, Inc. Compositions and methods for non-parenteral delivery of oligonucleotides
US6242589B1 (en) 1998-07-14 2001-06-05 Isis Pharmaceuticals, Inc. Phosphorothioate oligonucleotides having modified internucleoside linkages
US6867294B1 (en) 1998-07-14 2005-03-15 Isis Pharmaceuticals, Inc. Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages
US6225293B1 (en) 1998-09-02 2001-05-01 Isis Pharmaceuticals, Inc. Methods and compounds for tracking the biodistribution of macromolecule-carrier combinations
US6077709A (en) 1998-09-29 2000-06-20 Isis Pharmaceuticals Inc. Antisense modulation of Survivin expression
CA2344653A1 (en) 1998-09-29 2000-04-06 Gamida Cell Ltd. Methods of controlling proliferation and differentiation of stem and progenitor cells
US6300320B1 (en) 1999-01-05 2001-10-09 Isis Pharmaceuticals, Inc. Modulation of c-jun using inhibitors of protein kinase C
US6127124A (en) 1999-01-20 2000-10-03 Isis Pharmaceuticals, Inc. Fluorescence based nuclease assay
US7098192B2 (en) 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
US7534605B2 (en) * 1999-06-08 2009-05-19 Yissum Research Development Company Of The Hebrew University Of Jerusalem CD44 polypeptides, polynucleotides encoding same, antibodies directed thereagainst and method of using same for diagnosing and treating inflammatory diseases
US6656730B1 (en) 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
US6593466B1 (en) 1999-07-07 2003-07-15 Isis Pharmaceuticals, Inc. Guanidinium functionalized nucleotides and precursors thereof
US7332275B2 (en) 1999-10-13 2008-02-19 Sequenom, Inc. Methods for detecting methylated nucleotides
US6261840B1 (en) 2000-01-18 2001-07-17 Isis Pharmaceuticals, Inc. Antisense modulation of PTP1B expression
US20020055479A1 (en) 2000-01-18 2002-05-09 Cowsert Lex M. Antisense modulation of PTP1B expression
WO2001079284A2 (en) * 2000-04-13 2001-10-25 Wight Thomas N Therapeutic compounds and methods for formulating v3, a versican isoform
US6680172B1 (en) 2000-05-16 2004-01-20 Regents Of The University Of Michigan Treatments and markers for cancers of the central nervous system
US6656700B2 (en) 2000-05-26 2003-12-02 Amersham Plc Isoforms of human pregnancy-associated protein-E
US6686188B2 (en) 2000-05-26 2004-02-03 Amersham Plc Polynucleotide encoding a human myosin-like polypeptide expressed predominantly in heart and muscle
US6958214B2 (en) 2000-07-10 2005-10-25 Sequenom, Inc. Polymorphic kinase anchor proteins and nucleic acids encoding the same
US8568766B2 (en) 2000-08-24 2013-10-29 Gattadahalli M. Anantharamaiah Peptides and peptide mimetics to treat pathologies associated with eye disease
DE10046184A1 (de) * 2000-09-18 2002-04-04 November Ag Molekulare Medizin Verfahren zum Nachweis mindestens einer Nukleinsäuresequenz
CA2425779C (en) 2000-10-12 2013-08-06 University Of Rochester Compositions that inhibit proliferation of cancer cells
US7767802B2 (en) 2001-01-09 2010-08-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of anti-apoptotic genes
US6573051B2 (en) * 2001-03-09 2003-06-03 Molecular Staging, Inc. Open circle probes with intramolecular stem structures
WO2002072790A2 (en) 2001-03-14 2002-09-19 Myriad Genetics, Inc Tsg101-gag interaction and use thereof
EP2000148A1 (en) 2001-06-20 2008-12-10 Genentech, Inc. Compositions and methods for the diagnosis and treatment of prostate cancer
DK2423335T3 (da) 2001-06-21 2014-08-18 Dynavax Tech Corp Kimæriske immunmodulatoriske forbindelser og fremgangsmåder til anvendelse deraf
CA2451643C (en) 2001-06-21 2012-11-13 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
US7785610B2 (en) * 2001-06-21 2010-08-31 Dynavax Technologies Corporation Chimeric immunomodulatory compounds and methods of using the same—III
JP2005504020A (ja) * 2001-07-03 2005-02-10 アイシス・ファーマシューティカルス・インコーポレーテッド ヌクレアーゼ耐性キメラオリゴヌクレオチド
US7425545B2 (en) 2001-07-25 2008-09-16 Isis Pharmaceuticals, Inc. Modulation of C-reactive protein expression
US6964950B2 (en) 2001-07-25 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of C-reactive protein expression
US20030096772A1 (en) 2001-07-30 2003-05-22 Crooke Rosanne M. Antisense modulation of acyl CoA cholesterol acyltransferase-2 expression
US7407943B2 (en) 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
US20040096880A1 (en) * 2001-08-07 2004-05-20 Kmiec Eric B. Compositions and methods for the treatment of diseases exhibiting protein misassembly and aggregation
EP1423537A4 (en) * 2001-08-07 2006-11-29 Univ Delaware COMPOSITIONS AND METHODS FOR PREVENTING AND TREATING HUNTINGTON'S DISEASE
US7227014B2 (en) 2001-08-07 2007-06-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein (a) expression
EP2153843B1 (en) 2001-09-18 2012-08-15 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
US7070933B2 (en) * 2001-09-28 2006-07-04 Gen-Probe Incorporated Inversion probes
US6750019B2 (en) 2001-10-09 2004-06-15 Isis Pharmaceuticals, Inc. Antisense modulation of insulin-like growth factor binding protein 5 expression
ATE516364T1 (de) 2001-10-09 2011-07-15 Isis Pharmaceuticals Inc Antisense-modulation der expression des insulinähnlicher-wachstumsfaktor-bindungsprotei s 5
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
MXPA04006554A (es) 2002-01-02 2005-03-31 Genentech Inc Composiciones y metodos para diagnostico y tratamiento de tumor.
IL152904A0 (en) 2002-01-24 2003-06-24 Gamida Cell Ltd Utilization of retinoid and vitamin d receptor antagonists for expansion of renewable stem cell populations
EP1465982A4 (en) 2002-01-25 2006-06-07 Gamida Cell Ltd PROCESS FOR EXPANSION OF STEM AND PRESERVATIVE CELLS AND EXPANDED CELL POPULATIONS THEREWITH OBTAINED
CA2474709A1 (en) * 2002-02-04 2003-08-14 Biomira, Inc. Immunostimulatory, covalently lipidated oligonucleotides
US7553619B2 (en) 2002-02-08 2009-06-30 Qiagen Gmbh Detection method using dissociated rolling circle amplification
US20030180712A1 (en) 2002-03-20 2003-09-25 Biostratum Ab Inhibition of the beta3 subunit of L-type Ca2+ channels
KR20040101502A (ko) 2002-04-16 2004-12-02 제넨테크, 인크. 종양의 진단 및 치료 방법 및 이를 위한 조성물
US7432342B2 (en) 2002-05-03 2008-10-07 Sequenom, Inc. Kinase anchor protein muteins, peptides thereof and related documents
US7176181B2 (en) * 2002-05-21 2007-02-13 Yeda Research And Development Co. Ltd. Compositions and methods of using galectin-8 as an inhibitor of tumor cell growth
US7199107B2 (en) 2002-05-23 2007-04-03 Isis Pharmaceuticals, Inc. Antisense modulation of kinesin-like 1 expression
US20050221326A1 (en) * 2002-06-12 2005-10-06 Avi Orr-Urtreger Oligonucleotides antibodies and kits including same for treating prostate cancer and determining predisposition thereto
EP1575992A4 (en) 2002-08-05 2007-02-21 Univ Rochester CHEMICAL PROTEINS FROM PROTEIN TRANSDUCATING DOMAIN / DEAMINASE, RELATED COMPOUNDS AND THEIR USES
WO2004024919A1 (en) 2002-09-13 2004-03-25 Replicor, Inc. Non-sequence complementary antiviral oligonucleotides
JP2006500030A (ja) 2002-09-20 2006-01-05 イェール ユニバーシティ リボスイッチ、その使用方法、ならびにリボスイッチとともに用いるための組成物
WO2004031350A2 (en) 2002-09-26 2004-04-15 Amgen, Inc. Modulation of forkhead box o1a expression
US9150605B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
WO2004044138A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
WO2004044136A2 (en) 2002-11-05 2004-05-27 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2’-modified nucleosides for use in gene modulation
US9150606B2 (en) 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2'-modified nucleosides for use in gene modulation
EP2336318B1 (en) 2002-11-13 2013-04-24 Genzyme Corporation Antisense modulation of apolipoprotein b expression
DK1569695T3 (da) 2002-11-13 2013-08-05 Genzyme Corp Antisense-modulering af apolipoprotein-b-ekspression
US20060009378A1 (en) 2002-11-14 2006-01-12 Itshak Golan Novel galectin sequences and compositions and methods utilizing same for treating or diagnosing arthritis and other chronic inflammatory diseases
AU2003298650B2 (en) 2002-11-15 2010-03-11 Musc Foundation For Research Development Complement receptor 2 targeted complement modulators
ATE444359T1 (de) 2002-11-15 2009-10-15 Morphotek Inc Verfahren zur erzeugung einer hohen antikörperproduktion von durch in-vitro- immunisierung entstandenen hybridomen
EP2410332A1 (en) 2002-11-21 2012-01-25 The University Of Utah Method for identifying purinergic modulators of the olfactory system
US7144999B2 (en) 2002-11-23 2006-12-05 Isis Pharmaceuticals, Inc. Modulation of hypoxia-inducible factor 1 alpha expression
US20040110698A1 (en) * 2002-12-10 2004-06-10 Kimron Veterinary Institute Oligonucleotides and methods using same for treating cox-ll associated diseases
US20040115643A1 (en) * 2002-12-12 2004-06-17 Lizardi Paul M. Thermodynamic equilibrium extension of primers
US20040121338A1 (en) * 2002-12-19 2004-06-24 Alsmadi Osama A. Real-time detection of rolling circle amplification products
CA2510587A1 (en) 2002-12-20 2004-07-15 Qiagen Gmbh Nucleic acid amplification
US9487823B2 (en) 2002-12-20 2016-11-08 Qiagen Gmbh Nucleic acid amplification
US6977153B2 (en) 2002-12-31 2005-12-20 Qiagen Gmbh Rolling circle amplification of RNA
CA2515484C (en) 2003-02-11 2011-09-20 Antisense Therapeutics Ltd Modulation of insulin like growth factor i receptor expression
KR101130181B1 (ko) 2003-02-27 2012-03-28 예다 리서치 앤드 디벨럽먼트 캄파니 리미티드 인플루엔자 바이러스 감염의 치료 및 검출에 유용한 핵산분자, 폴리펩티드, 항체 및 이를 포함하는 조성물
US7803781B2 (en) 2003-02-28 2010-09-28 Isis Pharmaceuticals, Inc. Modulation of growth hormone receptor expression and insulin-like growth factor expression
US20040185559A1 (en) 2003-03-21 2004-09-23 Isis Pharmaceuticals Inc. Modulation of diacylglycerol acyltransferase 1 expression
US8043834B2 (en) 2003-03-31 2011-10-25 Qiagen Gmbh Universal reagents for rolling circle amplification and methods of use
US7598227B2 (en) 2003-04-16 2009-10-06 Isis Pharmaceuticals Inc. Modulation of apolipoprotein C-III expression
US7399853B2 (en) 2003-04-28 2008-07-15 Isis Pharmaceuticals Modulation of glucagon receptor expression
WO2004108081A2 (en) 2003-06-02 2004-12-16 Isis Pharmaceuticals, Inc. Oligonucleotide synthesis with alternative solvents
CA2524495A1 (en) 2003-06-03 2005-01-13 Eli Lilly And Company Modulation of survivin expression
US20040248103A1 (en) * 2003-06-04 2004-12-09 Feaver William John Proximity-mediated rolling circle amplification
ES2761262T3 (es) 2003-06-13 2020-05-19 Alnylam Europe Ag Acido ribonucleico bicatenario con elevada eficacia en un organismo
EP1648914A4 (en) 2003-07-31 2009-12-16 Regulus Therapeutics Inc OLIGOMERIC COMPOUNDS AND COMPOSITIONS USEFUL FOR MODULATING SMALL NON-CODING RNA
US7825235B2 (en) 2003-08-18 2010-11-02 Isis Pharmaceuticals, Inc. Modulation of diacylglycerol acyltransferase 2 expression
US20050053981A1 (en) 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
BRPI0413930A (pt) 2003-09-18 2006-10-24 Isis Pharmaceuticals Inc composto oligomérico ou sal farmaceuticamente aceitável deste, composição farmacêutica ou veterinária, métodos para inibir a expressão de eif4e em uma célula, tecido ou órgão, para diminuir a proliferação de uma célula em que eif4e é expressado, e para prevenir ou tratar uma condição ou doença métodos para prevenir ou diminuir a angiogênese, e o crescimento de tumor em um paciente, oligonucleotìdeo de anti-sentido, composição farmacêutica ou veterinária, e, uso de um composto oligomérico ou sal farmaceuticamente aceitável deste
JP5379347B2 (ja) 2003-09-18 2013-12-25 アイシス ファーマシューティカルズ， インコーポレーテッド ４’−チオヌクレオシドおよびオリゴマー化合物
US20050191653A1 (en) 2003-11-03 2005-09-01 Freier Susan M. Modulation of SGLT2 expression
ES2472690T3 (es) 2003-11-17 2014-07-02 Genentech, Inc. Anticuerpo contra CD22 para el tratamiento de tumores de origen hematopoy�tico
WO2006054296A2 (en) 2004-11-17 2006-05-26 Spectrum Dynamics Llc Methods of detecting prostate cancer
EP2363480A3 (en) 2004-01-20 2015-10-07 Isis Pharmaceuticals, Inc. Modulation of glucocorticoid receptor expression
US7468431B2 (en) 2004-01-22 2008-12-23 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP2 expression
US8778900B2 (en) 2004-01-22 2014-07-15 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP1 expression
US7842459B2 (en) 2004-01-27 2010-11-30 Compugen Ltd. Nucleotide and amino acid sequences, and assays and methods of use thereof for diagnosis
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
WO2005089268A2 (en) 2004-03-15 2005-09-29 Isis Pharmaceuticals, Inc. Compositions and methods for optimizing cleavage of rna by rnase h
WO2005097817A2 (en) 2004-04-05 2005-10-20 Alnylam Pharmaceuticals, Inc. Process and reagents for oligonucleotide synthesis and purification
US20050244869A1 (en) 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
EP3034510A1 (en) 2004-04-30 2016-06-22 Alnylam Pharmaceuticals Inc. Oligonucleotides comprising a c5-modified pyrimidine
NZ550772A (en) 2004-05-21 2009-10-30 Uab Research Foundation Variable lymphocyte receptors, related polypeptides and nucleic acids, and uses thereof
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
WO2006030442A2 (en) 2004-09-16 2006-03-23 Gamida-Cell Ltd. Methods of ex vivo progenitor and stem cell expansion by co-culture with mesenchymal cells
CA2989941A1 (en) 2004-09-23 2006-03-30 Arc Pharmaceuticals, Inc. Pharmaceutical compostions and methods relating to inhibiting fibrous adhesions or inflammatory disease using low sulphate fucans
CA2588087A1 (en) * 2004-11-15 2006-05-18 Obe Therapy Biotechnology S.A.S. Methods of reducing body fat
CA2597845A1 (en) * 2005-02-25 2006-08-31 Isis Pharmaceuticals, Inc. Compositions and their uses directed to il-4r alpha
EP2050763A3 (en) 2005-03-10 2009-07-15 Genentech, Inc. Methods and compositions for modulating vascular integrity
US7476733B2 (en) 2005-03-25 2009-01-13 The United States Of America As Represented By The Department Of Health And Human Services Development of a real-time PCR assay for detection of pneumococcal DNA and diagnosis of pneumococccal disease
EP1863908B1 (de) 2005-04-01 2010-11-17 Qiagen GmbH Reverse transkription und amplifikation von rna bei simultaner degradierung von dna
EP1891141B1 (en) 2005-05-31 2016-11-16 Ecole Polytechnique Fédérale de Lausanne (EPFL) Triblock copolymers for cytoplasmic delivery of gene-based drugs
US20090209621A1 (en) 2005-06-03 2009-08-20 The Johns Hopkins University Compositions and methods for decreasing microrna expression for the treatment of neoplasia
WO2006138145A1 (en) 2005-06-14 2006-12-28 Northwestern University Nucleic acid functionalized nanoparticles for therapeutic applications
EP1915448B1 (en) 2005-07-07 2013-09-04 Yissum Research Development Company, of The Hebrew University of Jerusalem Nucleic acid agents for downregulating h19, and methods of using same
ATE483802T1 (de) 2005-08-11 2010-10-15 Synthetic Genomics Inc Verfahren zur in vitro-rekombination
EP1915461B1 (en) 2005-08-17 2018-08-01 Dx4U GmbH Composition and method for determination of ck19 expression
EP2338991B1 (en) 2005-08-29 2017-01-18 Regulus Therapeutics Inc. Methods for use in modulating MIR-122a
EP1931780B1 (en) 2005-08-29 2016-01-06 Regulus Therapeutics Inc. Antisense compounds having enhanced anti-microrna activity
EP1762627A1 (de) 2005-09-09 2007-03-14 Qiagen GmbH Verfahren zur Aktivierung einer Nukleinsäure für eine Polymerase-Reaktion
IL172297A (en) 2005-10-03 2016-03-31 Compugen Ltd Soluble vegfr-1 variants for the diagnosis of preeclampsia
CA2625891A1 (en) 2005-10-14 2007-04-26 Carlton D. Donald Inhibition of pax2 by defb1 induction as a therapy for cancer
US8080534B2 (en) 2005-10-14 2011-12-20 Phigenix, Inc Targeting PAX2 for the treatment of breast cancer
JP5111385B2 (ja) 2005-10-28 2013-01-09 アルナイラム ファーマシューティカルズ， インコーポレイテッド ハンチンチン遺伝子の発現を抑制するための組成物および方法
ATE510545T1 (de) 2005-11-09 2011-06-15 Alnylam Pharmaceuticals Inc Zusammensetzungen und verfahren zur hemmung der expression von faktor v leiden mutationsgen
JP2009516710A (ja) 2005-11-21 2009-04-23 アイシス ファーマシューティカルズ， インコーポレーテッド ｅＩＦ４Ｅ−ＢＰ２の発現のモジュレート
US8846393B2 (en) 2005-11-29 2014-09-30 Gamida-Cell Ltd. Methods of improving stem cell homing and engraftment
WO2007076366A2 (en) * 2005-12-20 2007-07-05 Isis Pharmaceuticals, Inc Double stranded nucleic acid molecules targeted to il-4 receptor alpha
WO2007100412A2 (en) * 2005-12-21 2007-09-07 Yale University Methods and compositions related to the modulation of riboswitches
EP1976567B1 (en) 2005-12-28 2020-05-13 The Scripps Research Institute Natural antisense and non-coding rna transcripts as drug targets
EP2216339A1 (en) 2006-01-16 2010-08-11 Compugen Ltd. Novel nucleotide and amino acid sequences, and methods of use thereof for diagnosis
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
AU2007211082B2 (en) 2006-01-27 2012-09-27 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of microRNAs
JP5342881B2 (ja) 2006-01-27 2013-11-13 アイシス ファーマシューティカルズ， インコーポレーテッド ６−修飾された二環式核酸類似体
CN101448849B (zh) 2006-03-31 2013-08-21 阿尔尼拉姆医药品有限公司 抑制Eg5基因表达的组合物和方法
CA2651031A1 (en) 2006-05-03 2007-11-08 Baltic Technology Development, Ltd. Antisense agents combining strongly bound base - modified oligonucleotide and artificial nuclease
EP2505646A1 (en) 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of CRP
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
WO2007134161A2 (en) 2006-05-11 2007-11-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the pcsk9 gene
US7547684B2 (en) 2006-05-11 2009-06-16 Isis Pharmaceuticals, Inc. 5′-modified bicyclic nucleic acid analogs
ATE528008T1 (de) 2006-05-19 2011-10-15 Alnylam Pharmaceuticals Inc Rnai-modulation von aha und ihre therapeutische verwendung
AU2007253677B2 (en) 2006-05-22 2011-02-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of IKK-B gene
WO2008011473A2 (en) 2006-07-19 2008-01-24 Isis Pharmaceuticals, Inc. Compositions and their uses directed to hbxip
WO2008033866A2 (en) * 2006-09-11 2008-03-20 Yale University Methods and compositions for the use of lysine riboswitches
AU2007299629C1 (en) 2006-09-21 2012-05-10 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the HAMP gene
MX2009003548A (es) 2006-10-03 2009-04-15 Alnylam Pharmaceuticals Inc Formulaciones que contienen lipidos.
WO2008067040A2 (en) 2006-10-06 2008-06-05 University Of Utah Research Foundation Method of detecting ocular diseases and pathologic conditions and treatment of same
US8999317B2 (en) 2006-11-01 2015-04-07 University Of Rochester Methods and compositions related to the structure and function of APOBEC3G
US8093222B2 (en) 2006-11-27 2012-01-10 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
EP2455471A3 (en) * 2006-11-27 2012-09-12 Isis Pharmaceuticals, Inc. Methods for treating hypercholesterolemia
CA2672297A1 (en) 2006-12-11 2008-06-19 University Of Utah Research Foundation Compositions and methods for treating pathologic angiogenesis and vascular permeability
CN102604951A (zh) * 2006-12-14 2012-07-25 诺瓦提斯公司 治疗肌肉和心血管病症的组合物和方法
EP2913341A1 (en) 2006-12-22 2015-09-02 University of Utah Research Foundation Method of detecting ocular diseases and pathologic conditions and treatment of same
WO2008087641A2 (en) * 2007-01-16 2008-07-24 Yissum Research Development Company Of The Hebrew University Of Jerusalem H19 silencing nucleic acid agents for treating rheumatoid arthritis
WO2008093331A1 (en) * 2007-01-29 2008-08-07 Yissum Research Development Company Of The Hebrew University Of Jerusalem Antibody conjugates for circumventing multi-drug resistance
WO2009045469A2 (en) 2007-10-02 2009-04-09 Amgen Inc. Increasing erythropoietin using nucleic acids hybridizable to micro-rna and precursors thereof
CA2691066C (en) 2007-02-09 2018-07-31 Northwestern University Particles for detecting intracellular targets
EP2129803A4 (en) * 2007-02-27 2010-11-03 Univ Northwestern APPLICATION OF MOLECULES TO NANOPARTICLES
JP2010521977A (ja) 2007-03-22 2010-07-01 イェール ユニバーシティー オルタナティブスプライシングを調節するリボスイッチに関する方法および組成物
PE20090064A1 (es) 2007-03-26 2009-03-02 Novartis Ag Acido ribonucleico de doble cadena para inhibir la expresion del gen e6ap humano y composicion farmaceutica que lo comprende
JP5350360B2 (ja) 2007-03-29 2013-11-27 アルナイラム ファーマシューティカルズ， インコーポレイテッド エボラ由来の遺伝子の発現を阻害するための組成物および方法
EP2162552A4 (en) 2007-05-11 2010-06-30 Univ Johns Hopkins BIOMARKER FOR MELANOME
WO2008156987A2 (en) 2007-05-29 2008-12-24 Yale University Riboswitches and methods and compositions for use of and with riboswitches
CA2687684A1 (en) * 2007-05-29 2008-12-11 Yale University Methods and compositions related to riboswitches that control alternative splicing and rna processing
EP2170917B1 (en) 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
EP2160464B1 (en) 2007-05-30 2014-05-21 Northwestern University Nucleic acid functionalized nanoparticles for therapeutic applications
US7807372B2 (en) * 2007-06-04 2010-10-05 Northwestern University Screening sequence selectivity of oligonucleotide-binding molecules using nanoparticle based colorimetric assay
EP2173760B2 (en) 2007-06-08 2015-11-04 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
EP2319926B1 (en) 2007-07-05 2016-08-31 Arrowhead Research Corporation DSRNA for treating viral infection
CA2692579C (en) 2007-07-05 2016-05-03 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
AU2008286771B2 (en) 2007-08-15 2013-08-15 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
US8557767B2 (en) 2007-08-28 2013-10-15 Uab Research Foundation Synthetic apolipoprotein E mimicking polypeptides and methods of use
WO2009032693A2 (en) 2007-08-28 2009-03-12 Uab Research Foundation Synthetic apolipoprotein e mimicking polypeptides and methods of use
EP2769729B1 (en) 2007-09-04 2019-01-09 Compugen Ltd. Polypeptides and polynucleotides, and uses thereof as a drug target for producing drugs and biologics
US8445217B2 (en) 2007-09-20 2013-05-21 Vanderbilt University Free solution measurement of molecular interactions by backscattering interferometry
US7951785B2 (en) 2007-09-21 2011-05-31 California Institute Of Technology NFIA in glial fate determination, glioma therapy and astrocytoma treatment
US8097712B2 (en) 2007-11-07 2012-01-17 Beelogics Inc. Compositions for conferring tolerance to viral disease in social insects, and the use thereof
EP2222851B1 (en) 2007-11-20 2017-06-28 Ionis Pharmaceuticals, Inc. Modulation of cd40 expression
WO2009067647A1 (en) * 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
EP2617828B1 (en) 2007-12-10 2014-09-24 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of factor VII gene
WO2009086558A1 (en) 2008-01-02 2009-07-09 Tekmira Pharmaceuticals Corporation Improved compositions and methods for the delivery of nucleic acids
EP2265627A2 (en) 2008-02-07 2010-12-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
BRPI0909779A2 (pt) 2008-03-05 2019-09-24 Alnylam Pharmaceuticals Inc composições e processos para a inibição da expressão dos genes eg5 e vegf
US8426378B2 (en) 2008-03-21 2013-04-23 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising tricyclic nucelosides and methods for their use
EP2982753B1 (en) 2008-04-18 2018-06-06 Baxter International Inc. Microsphere-based composition for preventing and/or reversing new-onset autoimmune diabetes
WO2009134917A2 (en) * 2008-04-29 2009-11-05 Wyeth Methods for treating inflammation
US8341614B2 (en) * 2008-06-06 2012-12-25 Apple Inc. Memory management for closures
KR101877698B1 (ko) 2008-08-25 2018-07-12 엑스칼리아드 파마슈티컬즈, 인코포레이티드 결합 조직 성장 인자에 대한 안티센스 올리고뉴클레오타이드 및 그의 용도
WO2010028054A1 (en) 2008-09-02 2010-03-11 Alnylam Europe Ag. Compositions and methods for inhibiting expression of mutant egfr gene
WO2010036698A1 (en) 2008-09-24 2010-04-01 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
EP2361256B1 (en) 2008-09-24 2013-04-10 Isis Pharmaceuticals, Inc. Cyclohexenyl nucleic acid analogs
EP3584320A1 (en) 2008-09-25 2019-12-25 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of serum amyloid a gene
EP2743265B1 (en) 2008-10-09 2017-03-15 Arbutus Biopharma Corporation Improved amino lipids and methods for the delivery of nucleic acids
AU2009305636A1 (en) 2008-10-15 2010-04-22 Ionis Pharmaceuticals, Inc. Modulation of Factor 11 expression
PT2937418T (pt) 2008-10-20 2018-01-23 Alnylam Pharmaceuticals Inc Composições e métodos de inibição da expressão de transtirretina
US20120059045A1 (en) 2008-10-24 2012-03-08 Isis Pharmaceuticals, Inc. Methods of using oligomeric compounds comprising 2'-substituted nucleosides
EP2358398A2 (en) 2008-10-24 2011-08-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
AU2009316286B2 (en) 2008-11-24 2016-05-26 Northwestern University Polyvalent RNA-nanoparticle compositions
WO2010061393A1 (en) 2008-11-30 2010-06-03 Compugen Ltd. He4 variant nucleotide and amino acid sequences, and methods of use thereof
WO2010065792A2 (en) 2008-12-04 2010-06-10 Curna, Inc. Treatment of erythropoietin (epo) related diseases by inhibition of natural antisense transcript to epo
KR20110091796A (ko) 2008-12-04 2011-08-12 오피케이오 큐알엔에이, 엘엘씨 종양 억제 유전자에 대한 천연 안티센스 전사체의 억제에 의해 종양 억제 유전자 관련된 질환의 치료
CN108042560A (zh) 2008-12-04 2018-05-18 库尔纳公司 通过抑制针对沉默调节蛋白1的天然反义转录物来治疗沉默调节蛋白1相关的疾病
WO2010064248A2 (en) 2008-12-05 2010-06-10 Yeda Research And Development Co. Ltd. Methods of diagnosing and treating motor neuron diseases
NZ593618A (en) 2008-12-10 2013-02-22 Alnylam Pharmaceuticals Inc Gnaq targeted dsrna compositions and methods for inhibiting expression
WO2010080554A1 (en) 2008-12-17 2010-07-15 Avi Biopharma, Inc. Antisense compositions and methods for modulating contact hypersensitivity or contact dermatitis
US20100233270A1 (en) 2009-01-08 2010-09-16 Northwestern University Delivery of Oligonucleotide-Functionalized Nanoparticles
EP2391343B1 (en) 2009-01-29 2017-03-01 Arbutus Biopharma Corporation Improved lipid formulation for the delivery of nucleic acids
US9745574B2 (en) 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
US8536320B2 (en) 2009-02-06 2013-09-17 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
CA2752239C (en) 2009-02-12 2021-03-30 Opko Curna, Llc Treatment of glial cell derived neurotrophic factor (gdnf) related diseases by inhibition of natural antisense transcript to gdnf
EP2396038B1 (en) 2009-02-12 2015-10-21 CuRNA, Inc. Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
WO2010099341A1 (en) 2009-02-26 2010-09-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of mig-12 gene
EP2669290A1 (en) 2009-03-02 2013-12-04 Alnylam Pharmaceuticals Inc. Nucleic Acid Chemical Modifications
US20110319317A1 (en) 2009-03-04 2011-12-29 Opko Curna, Llc Treatment of sirtuin 1 (sirt1) related diseases by inhibition of natural antisense transcript to sirt1
NZ594995A (en) 2009-03-12 2013-06-28 Alnylam Pharmaceuticals Inc LIPID FORMULATED COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF HUMAN KINESIN FAMILY MEMBER 11 (Eg5) AND VASCULAR ENDOTHELIAL GROWTH FACTOR (VEGF) GENES
CN102482677B (zh) 2009-03-16 2017-10-17 库尔纳公司 通过抑制nrf2的天然反义转录物治疗核因子(红细胞衍生2)‑样2(nrf2)相关疾病
EP2408920B1 (en) 2009-03-17 2017-03-08 CuRNA, Inc. Treatment of delta-like 1 homolog (dlk1) related diseases by inhibition of natural antisense transcript to dlk1
KR20120022938A (ko) 2009-04-15 2012-03-12 노오쓰웨스턴 유니버시티 올리고뉴클레오티드 관능화된 나노입자의 전달
EP3248618A1 (en) 2009-04-22 2017-11-29 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long rna molecules
US8318690B2 (en) 2009-05-01 2012-11-27 Curna, Inc. Treatment of hemoglobin (HBF/HBG) related diseases by inhibition of natural antisense transcript to HBF/HBG
AU2010245933B2 (en) 2009-05-05 2016-06-16 Arbutus Biopharma Corporation Methods of delivering oligonucleotides to immune cells
CA3042927C (en) 2009-05-05 2022-05-17 Arbutus Biopharma Corporation Lipid compositions for the delivery of therapeutic agents
CA2761152A1 (en) 2009-05-06 2010-11-11 Opko Curna, Llc Treatment of lipid transport and metabolism gene related diseases by inhibition of natural antisense transcript to a lipid transport and metabolism gene
US20120046236A1 (en) 2009-05-06 2012-02-23 Opko Curna Llc Treatment of tristetraproline (ttp) related diseases by inhibition of natural antisense transcript to ttp
US20120107331A1 (en) 2009-05-15 2012-05-03 Yale University Gemm riboswitches, structure-based compound design with gemm riboswitches, and methods and compositions for use of and with gemm riboswitches
CA2762369C (en) 2009-05-18 2021-12-28 Joseph Collard Treatment of reprogramming factor related diseases by inhibition of natural antisense transcript to a reprogramming factor
KR101703695B1 (ko) 2009-05-22 2017-02-08 큐알엔에이, 인크. 전사 인자 ｅ3(ｔｆｅ3)에 대한 천연 안티센스 전사체의 억제에 의해 ｔｆｅ3 및 인슐린 수용체 기질 2(ｉｒｓ2)의 치료
WO2010138806A2 (en) 2009-05-28 2010-12-02 Curna, Inc. Treatment of antiviral gene related diseases by inhibition of natural antisense transcript to an antiviral gene
HRP20211619T1 (hr) 2009-06-10 2022-02-04 Arbutus Biopharma Corporation Poboljšana formulacija lipida
EP2443237B1 (en) 2009-06-16 2017-02-22 CuRNA, Inc. Treatment of collagen gene related diseases by inhibition of natural antisense transcript to a collagen gene
KR101702689B1 (ko) 2009-06-16 2017-02-06 큐알엔에이, 인크. Ｐｏｎ1에 대한 천연 안티센스 전사체의 억제에 의한 파라옥소나제 1(ｐｏｎ1) 관련된 질환의 치료
CA2765889A1 (en) 2009-06-24 2010-12-29 Opko Curna, Llc Treatment of tumor necrosis factor receptor 2 (tnfr2) related diseases by inhibition of natural antisense transcript to tnfr2
US8921330B2 (en) 2009-06-26 2014-12-30 Curna, Inc. Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene
US9512164B2 (en) * 2009-07-07 2016-12-06 Alnylam Pharmaceuticals, Inc. Oligonucleotide end caps
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
ES2585360T3 (es) 2009-08-05 2016-10-05 Curna, Inc. Tratamiento de enfermedades relacionadas con un gen de la insulina (INS) por inhibición de la transcripción antisentido natural en un gen de la insulina (INS)
EP2462153B1 (en) 2009-08-06 2015-07-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
US9029338B2 (en) 2009-08-14 2015-05-12 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of a gene from the ebola virus
WO2011022420A1 (en) 2009-08-17 2011-02-24 Yale University Methylation biomarkers and methods of use
US9023822B2 (en) 2009-08-25 2015-05-05 Curna, Inc. Treatment of 'IQ motif containing GTPase activating protein' (IQGAP) related diseases by inhibition of natural antisense transcript to IQGAP
DK2473522T3 (en) 2009-09-02 2016-11-28 Genentech Inc Smoothened MUTANT AND METHODS OF USING THE SAME
US8962584B2 (en) 2009-10-14 2015-02-24 Yissum Research Development Company Of The Hebrew University Of Jerusalem, Ltd. Compositions for controlling Varroa mites in bees
EP3272869B1 (en) 2009-10-14 2020-06-24 Yissum Research Development Company of The Hebrew University of Jerusalem Ltd. Compositions for controlling varroa mites in bees
ES2564207T3 (es) 2009-10-22 2016-03-18 F. Hoffmann-La Roche Ag Métodos y composiciones para modular la activación con hepsina de la proteína estimuladora de macrófagos
WO2011056687A2 (en) 2009-10-27 2011-05-12 Swift Biosciences, Inc. Polynucleotide primers and probes
DK2494075T3 (en) 2009-10-30 2018-07-23 Univ Northwestern TABLE-MANAGED NANOCONJUGATES
KR20120102674A (ko) 2009-11-03 2012-09-18 유니버시티 오브 버지니아 페이턴트 파운데이션 중합체 분석물을 검출하는 다용도의 가시적인 방법
WO2011058555A1 (en) 2009-11-12 2011-05-19 Yeda Research And Development Co. Ltd. A method of editing dna in a cell and constructs capable of same
WO2011060320A1 (en) 2009-11-13 2011-05-19 Avi Biopharma, Inc. Antisense antiviral compound and method for treating influenza viral infection
AU2010321576A1 (en) 2009-11-23 2012-06-07 Swift Biosciences, Inc. Devices to extend single stranded target molecules
SG10201408598XA (en) 2009-11-30 2015-02-27 Genentech Inc Antibodies for treating and diagnosing tumors expressing slc34a2 (tat211 = seqid2 )
US9173895B2 (en) 2009-12-16 2015-11-03 Curna, Inc. Treatment of membrane bound transcription factor peptidase, site 1 (MBTPS1) related diseases by inhibition of natural antisense transcript to MBTPS1
RU2619185C2 (ru) 2009-12-23 2017-05-12 Курна, Инк. Лечение заболеваний, связанных с разобщающим белком 2 (ucp2), путем ингибирования природного антисмыслового транскрипта к ucp2
CN102869776B (zh) 2009-12-23 2017-06-23 库尔纳公司 通过抑制肝细胞生长因子(hgf)的天然反义转录物而治疗hgf相关疾病
US8962585B2 (en) 2009-12-29 2015-02-24 Curna, Inc. Treatment of tumor protein 63 (p63) related diseases by inhibition of natural antisense transcript to p63
EP2519633B1 (en) 2009-12-29 2017-10-25 CuRNA, Inc. Treatment of nuclear respiratory factor 1 (nrf1) related diseases by inhibition of natural antisense transcript to nrf1
NO2521784T3 (enExample) 2010-01-04 2018-05-05
EP2521785B1 (en) 2010-01-06 2022-03-09 CuRNA, Inc. Inhibition of natural antisense transcript to a pancreatic developmental gene for use in a treatment of pancreatic developmental gene related diseases
DK2524039T3 (en) 2010-01-11 2018-03-12 Curna Inc TREATMENT OF GENDER HORMON-BINDING GLOBULIN (SHBG) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPTS TO SHBG
US8779118B2 (en) 2010-01-11 2014-07-15 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
US20120321647A1 (en) 2010-01-12 2012-12-20 Yale University Structured rna motifs and compounds and methods for their use
DK2529015T3 (en) 2010-01-25 2018-02-26 Curna Inc TREATMENT OF RNASE H1-RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO RNASE H1
WO2011097407A1 (en) 2010-02-04 2011-08-11 Ico Therapeutics Inc. Dosing regimens for treating and preventing ocular disorders using c-raf antisense
WO2011103528A2 (en) 2010-02-22 2011-08-25 Opko Curna Llc Treatment of pyrroline-5-carboxylate reductase 1 (pycr1) related diseases by inhibition of natural antisense transcript to pycr1
WO2011105901A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 9 (c9) and uses thereof
WO2011105900A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-alpha (c8-alpha) and uses thereof
WO2011105902A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 8-beta (c8-beta) and uses thereof
WO2011106297A2 (en) 2010-02-23 2011-09-01 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
WO2011112516A1 (en) 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
SG183407A1 (en) 2010-03-08 2012-09-27 Monsanto Technology Llc Polynucleotide molecules for gene regulation in plants
US9068185B2 (en) 2010-03-12 2015-06-30 Sarepta Therapeutics, Inc. Antisense modulation of nuclear hormone receptors
WO2011113054A2 (en) 2010-03-12 2011-09-15 Aurasense Llc Crosslinked polynucleotide structure
ES2743600T3 (es) 2010-03-12 2020-02-20 Brigham & Womens Hospital Inc Métodos de tratamiento de los trastornos inflamatorios vasculares
US9193752B2 (en) 2010-03-17 2015-11-24 Isis Pharmaceuticals, Inc. 5′-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
US8889350B2 (en) 2010-03-26 2014-11-18 Swift Biosciences, Inc. Methods and compositions for isolating polynucleotides
ES2893199T3 (es) 2010-03-29 2022-02-08 Alnylam Pharmaceuticals Inc Terapia de ARNbc para amiloidosis ocular relacionada con transtiretina (TTR)
US9102938B2 (en) 2010-04-01 2015-08-11 Alnylam Pharmaceuticals, Inc. 2′ and 5′ modified monomers and oligonucleotides
EP4385568A3 (en) 2010-04-06 2025-02-12 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of cd274/pd-l1 gene
EP3517613A1 (en) 2010-04-09 2019-07-31 CuRNA, Inc. Treatment of fibroblast growth factor 21 (fgf21) related diseases by inhibition of natural antisense transcript to fgf21
WO2011133695A2 (en) 2010-04-20 2011-10-27 Swift Biosciences, Inc. Materials and methods for nucleic acid fractionation by solid phase entrapment and enzyme-mediated detachment
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
EP3091027B1 (en) 2010-04-28 2018-01-17 Ionis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
WO2011139702A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. Modified nucleosides and oligomeric compounds prepared therefrom
MX343559B (es) 2010-04-29 2016-11-10 Ionis Pharmaceuticals Inc Modulacion de la expresion de transtiretina.
CN107988228B (zh) 2010-05-03 2022-01-25 库尔纳公司 通过抑制沉默调节蛋白(sirt)的天然反义转录物而治疗沉默调节蛋白(sirt)相关疾病
BR112012028010A2 (pt) 2010-05-03 2017-09-26 Genentech Inc anticorpo isolado, célula, ácido nucleíco isolado, método de identificação de um primeiro anticorpo que se liga a um epítopo antigênico tat425 ligado or um anticorpo, métodos de inibir o crescimento de uma célula, de tratamento terapêutico de determinação da presença de uma proteína de tat425 e de diagnóstico da presença de um tumor em um mamífero
CA2805086C (en) 2010-05-13 2020-10-20 Sarepta Therapeutics, Inc. Antisense modulation of interleukins 17 and 23 signaling
TWI586356B (zh) 2010-05-14 2017-06-11 可娜公司 藉由抑制ｐａｒ４天然反股轉錄本治療ｐａｒ４相關疾病
CN102947451B (zh) 2010-05-26 2017-09-22 库尔纳公司 通过抑制无调同源物1(atoh1)的天然反义转录物而治疗atoh1相关疾病
WO2011150226A1 (en) 2010-05-26 2011-12-01 Landers James P Method for detecting nucleic acids based on aggregate formation
CA2801066C (en) 2010-06-02 2021-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
US8957200B2 (en) 2010-06-07 2015-02-17 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
US8846637B2 (en) 2010-06-08 2014-09-30 Isis Pharmaceuticals, Inc. Substituted 2′-amino and 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
US9638632B2 (en) 2010-06-11 2017-05-02 Vanderbilt University Multiplexed interferometric detection system and method
US9168297B2 (en) 2010-06-23 2015-10-27 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Regulation of skin pigmentation by neuregulin-1 (NRG-1)
US20130209463A1 (en) 2010-06-30 2013-08-15 Compugen Ltd. Polypeptides and uses thereof as a drug for treatment of multiple sclerosis, rheumatoid arthritis and other autoimmune disorders
US20130143955A1 (en) 2010-08-09 2013-06-06 Yale University Cyclic di-GMP-II Riboswitches, Motifs, and Compounds, and Methods for Their Use
US20130210901A1 (en) 2010-09-20 2013-08-15 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Method of treating neurodegenerative diseases
JP5974012B2 (ja) 2010-10-05 2016-08-23 ジェネンテック， インコーポレイテッド 突然変異体スムースンド及びその使用方法
JP5986998B2 (ja) 2010-10-06 2016-09-06 カッパーアールエヌエー，インコーポレイテッド シアリダーゼ４（ｎｅｕ４）への天然アンチセンス転写物の阻害によるｎｅｕ４関連疾患の治療
US20140031250A1 (en) 2010-10-07 2014-01-30 David Tsai Ting Biomarkers of Cancer
EP3075396A1 (en) 2010-10-17 2016-10-05 Yeda Research and Development Co. Ltd. Methods and compositions for the treatment of insulin-associated medical conditions
US9222088B2 (en) 2010-10-22 2015-12-29 Curna, Inc. Treatment of alpha-L-iduronidase (IDUA) related diseases by inhibition of natural antisense transcript to IDUA
DK2633052T3 (en) 2010-10-27 2018-07-16 Curna Inc TREATMENT OF INTERFERON-RELATED DEVELOPMENT REGULATOR 1 (IFRD1) -RELATED DISEASES BY INHIBITION OF NATURAL ANTISENCE TRANSCRIPT TO IFRD1
JP2012111933A (ja) * 2010-11-02 2012-06-14 Nitto Denko Corp 熱可塑性シリコーン樹脂
WO2012064824A1 (en) 2010-11-09 2012-05-18 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
EP3260540A1 (en) 2010-11-12 2017-12-27 The General Hospital Corporation Polycomb-associated non-coding rnas
EP3521451A1 (en) 2010-11-17 2019-08-07 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
CN103459599B (zh) 2010-11-23 2017-06-16 库尔纳公司 通过抑制nanog的天然反义转录物而治疗nanog相关疾病
US9150926B2 (en) 2010-12-06 2015-10-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Diagnosis and treatment of adrenocortical tumors using human microRNA-483
EP2649182A4 (en) 2010-12-10 2015-05-06 Alnylam Pharmaceuticals Inc Compositions and methods for increasing erythropoietin (epo) production
US9127275B2 (en) 2010-12-10 2015-09-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
US9045749B2 (en) 2011-01-14 2015-06-02 The General Hospital Corporation Methods targeting miR-128 for regulating cholesterol/lipid metabolism
EP2670404B1 (en) 2011-02-02 2018-08-29 The Trustees of Princeton University Sirtuin modulators as virus production modulators
EP2670411B1 (en) 2011-02-02 2019-04-10 Excaliard Pharmaceuticals, Inc. Antisense compounds targeting connective tissue growth factor (ctgf) for use in a method of treating keloids or hypertrophic scars
US9562853B2 (en) 2011-02-22 2017-02-07 Vanderbilt University Nonaqueous backscattering interferometric methods
EP3674409A1 (en) 2011-03-29 2020-07-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of tmprss6 gene
US10086043B2 (en) 2011-04-03 2018-10-02 The General Hospital Corporation Efficient protein expression in vivo using modified RNA (MOD-RNA)
RU2623161C2 (ru) 2011-04-15 2017-06-22 Компуджен Лтд. Полипептиды и полинуклеотиды и их применение для лечения иммунологических нарушений и рака
US20140186844A1 (en) 2011-04-26 2014-07-03 Swift Biosciences, Inc. Polynucleotide primers and probes
WO2012151289A2 (en) 2011-05-02 2012-11-08 University Of Virginia Patent Foundation Method and system to detect aggregate formation on a substrate
WO2012151268A1 (en) 2011-05-02 2012-11-08 University Of Virginia Patent Foundation Method and system for high throughput optical and label free detection of analytes
US9593330B2 (en) 2011-06-09 2017-03-14 Curna, Inc. Treatment of frataxin (FXN) related diseases by inhibition of natural antisense transcript to FXN
WO2012170347A1 (en) 2011-06-09 2012-12-13 Isis Pharmaceuticals, Inc. Bicyclic nucleosides and oligomeric compounds prepared therefrom
JP6110372B2 (ja) 2011-06-21 2017-04-05 アルナイラム ファーマシューティカルズ， インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． アンジオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物及びその使用方法
RU2631805C2 (ru) 2011-06-21 2017-09-26 Элнилэм Фармасьютикалз, Инк. Композиции и способы ингибирования экспрессии генов аполипопротеина с-iii (арос3)
WO2012177949A2 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein c (proc) genes
US20140235693A1 (en) 2011-06-23 2014-08-21 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
AU2012277376B2 (en) 2011-06-30 2016-11-24 Compugen Ltd. Polypeptides and uses thereof for treatment of autoimmune disorders and infection
WO2013019857A2 (en) 2011-08-01 2013-02-07 Alnylam Pharmaceuticals, Inc. Method for improving the success rate of hematopoietic stem cell transplants
WO2013018060A2 (en) 2011-08-04 2013-02-07 Yeda Research And Development Co. Ltd. Micro-rnas and compositions comprising same for the treatment and diagnosis of serotonin-, adrenalin-, noradrenalin-, glutamate-, and corticotropin-releasing hormone- associated medical conditions
US10760086B2 (en) 2011-09-13 2020-09-01 Monsanto Technology Llc Methods and compositions for weed control
US10806146B2 (en) 2011-09-13 2020-10-20 Monsanto Technology Llc Methods and compositions for weed control
US10829828B2 (en) 2011-09-13 2020-11-10 Monsanto Technology Llc Methods and compositions for weed control
AU2012308694B2 (en) 2011-09-13 2018-06-14 Monsanto Technology Llc Methods and compositions for weed control
CA2848680C (en) 2011-09-13 2020-05-19 Monsanto Technology Llc Methods and compositions for weed control
US9840715B1 (en) 2011-09-13 2017-12-12 Monsanto Technology Llc Methods and compositions for delaying senescence and improving disease tolerance and yield in plants
AU2012308686B2 (en) 2011-09-13 2018-05-10 Monsanto Technology Llc Methods and compositions for weed control
CA2848695A1 (en) 2011-09-13 2013-03-21 Monsanto Technology Llc Methods and composition for weed control comprising inhibiting ppg oxidase
CA2847698C (en) 2011-09-14 2020-09-01 Northwestern University Nanoconjugates able to cross the blood-brain barrier
US9920326B1 (en) 2011-09-14 2018-03-20 Monsanto Technology Llc Methods and compositions for increasing invertase activity in plants
EP3533873A1 (en) 2011-09-14 2019-09-04 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
WO2013040548A2 (en) 2011-09-17 2013-03-21 Yale University Fluoride-responsive riboswitchs, fluoride transporters, and methods of use
AU2012322788B2 (en) 2011-10-11 2018-01-04 The Brigham And Women's Hospital, Inc. Micrornas in neurodegenerative disorders
KR102102862B1 (ko) 2011-10-14 2020-04-22 제넨테크, 인크. 항-HtrA1 항체 및 사용 방법
EP2771464B1 (en) 2011-10-27 2018-03-21 Yeda Research and Development Co. Ltd. Methods of treating cancer
CA2895077A1 (en) 2011-12-12 2013-06-20 Beverly Packard In vivo delivery of oligonucleotides
WO2013106358A1 (en) 2012-01-10 2013-07-18 Hussain M Mahmood Method of treating hyperlipidemia and atherosclerosis with mir-30c
JP2015512616A (ja) 2012-02-01 2015-04-30 コンピュゲン エルティーディー． Ｃ１ｏｒｆ３２抗体およびがんの治療のためのその使用
JP6348848B2 (ja) 2012-02-13 2018-06-27 ガミダ セル リミテッド 間葉系幹細胞の増殖
EP2844745A2 (en) 2012-02-22 2015-03-11 Brainstem Biotec Ltd. Generation of neural stem cells and motor neurons
EP3401393B1 (en) 2012-02-22 2020-02-19 Exostem Biotec Ltd Micrornas for the generation of astrocytes
SG11201405669XA (en) 2012-03-13 2014-10-30 Swift Biosciences Inc Methods and compositions for size-controlled homopolymer tailing of substrate polynucleotides by a nucleic acid polymerase
EP2639238A1 (en) 2012-03-15 2013-09-18 Universität Bern Tricyclic nucleosides and oligomeric compounds prepared therefrom
EP2825648B1 (en) 2012-03-15 2018-09-05 CuRNA, Inc. Treatment of brain derived neurotrophic factor (bdnf) related diseases by inhibition of natural antisense transcript to bdnf
WO2013154799A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleosides and oligomeric compounds prepared therefrom
EP2850092B1 (en) 2012-04-09 2017-03-01 Ionis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
EP2839005B1 (en) 2012-04-20 2021-01-06 Aptamir Therapeutics, Inc. Mirna modulators of thermogenesis
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
WO2013163628A2 (en) 2012-04-27 2013-10-31 Duke University Genetic correction of mutated genes
US9273949B2 (en) 2012-05-11 2016-03-01 Vanderbilt University Backscattering interferometric methods
DK2850189T3 (en) 2012-05-16 2019-02-25 Translate Bio Ma Inc COMPOSITIONS AND PROCEDURES FOR MODULATING GENEPRESSION
DK2850186T3 (en) 2012-05-16 2019-04-08 Translate Bio Ma Inc COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY EXPRESSION
CA2873828A1 (en) 2012-05-24 2013-11-28 A.B. Seeds Ltd. Naked dsrna for silencing target molecules in plant seeds
WO2013184209A1 (en) 2012-06-04 2013-12-12 Ludwig Institute For Cancer Research Ltd. Mif for use in methods of treating subjects with a neurodegenerative disorder
US20140038182A1 (en) 2012-07-17 2014-02-06 Dna Logix, Inc. Cooperative primers, probes, and applications thereof
US9567569B2 (en) 2012-07-23 2017-02-14 Gamida Cell Ltd. Methods of culturing and expanding mesenchymal stem cells
US9175266B2 (en) 2012-07-23 2015-11-03 Gamida Cell Ltd. Enhancement of natural killer (NK) cell proliferation and activity
CA2880833A1 (en) 2012-08-03 2014-02-06 Aptamir Therapeutics, Inc. Cell-specific delivery of mirna modulators for the treatment of obesity and related disorders
WO2014028739A1 (en) 2012-08-15 2014-02-20 Isis Pharmaceuticals, Inc. Method of preparing oligomeric compounds using modified capping protocols
CA2880869A1 (en) 2012-08-20 2014-02-27 The Regents Of The University Of California Polynucleotides having bioreversible groups
US9029335B2 (en) 2012-10-16 2015-05-12 Isis Pharmaceuticals, Inc. Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
MX364070B (es) 2012-10-18 2019-04-10 Monsanto Technology Llc Métodos y composiciones para el control de plagas vegetales.
HK1214830A1 (zh) 2012-11-05 2016-08-05 Foundation Medicine, Inc. 新型ntrk1融合分子及其应用
US11230589B2 (en) 2012-11-05 2022-01-25 Foundation Medicine, Inc. Fusion molecules and uses thereof
EP2941487A2 (en) 2013-01-01 2015-11-11 A.B. Seeds Ltd. ISOLATED dsRNA MOLECULES AND METHODS OF USING SAME FOR SILENCING TARGET MOLECULES OF INTEREST
US10683505B2 (en) 2013-01-01 2020-06-16 Monsanto Technology Llc Methods of introducing dsRNA to plant seeds for modulating gene expression
AU2014207342C1 (en) 2013-01-18 2019-04-04 Foundation Medicine, Inc. Methods of treating cholangiocarcinoma
US10000767B2 (en) 2013-01-28 2018-06-19 Monsanto Technology Llc Methods and compositions for plant pest control
WO2014120861A2 (en) 2013-01-31 2014-08-07 Isis Pharmaceuticals, Inc. Method of preparing oligomeric compounds using modified coupling protocols
WO2014130922A1 (en) 2013-02-25 2014-08-28 Trustees Of Boston University Compositions and methods for treating fungal infections
CN105074008A (zh) 2013-03-13 2015-11-18 孟山都技术有限公司 用于杂草控制的方法和组合物
CN105263329B (zh) 2013-03-13 2020-09-18 孟山都技术公司 用于杂草控制的方法和组合物
US20140283211A1 (en) 2013-03-14 2014-09-18 Monsanto Technology Llc Methods and Compositions for Plant Pest Control
EP2970364B1 (en) 2013-03-14 2019-04-24 Andes Biotechnologies Global, Inc. Methods for detecting and treating multiple myeloma
PL2970974T3 (pl) 2013-03-14 2018-01-31 Alnylam Pharmaceuticals Inc Kompozycje komponentu C5 dopełniacza iRNA i sposoby ich zastosowania
PL2970970T3 (pl) 2013-03-14 2019-06-28 Andes Biotechnologies Global, Inc. Oligonukleotydy antysensowe do leczenia nowotworowych komórek macierzystych
US10568328B2 (en) 2013-03-15 2020-02-25 Monsanto Technology Llc Methods and compositions for weed control
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
WO2014165825A2 (en) 2013-04-04 2014-10-09 President And Fellows Of Harvard College Therapeutic uses of genome editing with crispr/cas systems
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
LT2999785T (lt) 2013-05-22 2018-07-25 Alnylam Pharmaceuticals, Inc. Serpina1 irna kompozicijos ir jų panaudojimo būdai
BR112015029276B1 (pt) 2013-05-22 2022-07-12 Alnylam Pharmaceuticals, Inc Agente de irna fita dupla capaz de inibir a expressão de tmprss6, composição farmacêutica e uso dos mesmos
EP3004396B1 (en) 2013-06-06 2019-10-16 The General Hospital Corporation Compositions for the treatment of cancer
WO2014197938A1 (en) 2013-06-13 2014-12-18 Antisense Therapeutics Ltd Combination therapy
US9850496B2 (en) 2013-07-19 2017-12-26 Monsanto Technology Llc Compositions and methods for controlling Leptinotarsa
UA122662C2 (uk) 2013-07-19 2020-12-28 Монсанто Текнолоджі Ллс Композиція та спосіб боротьби з leptinotarsa
KR20160042917A (ko) 2013-07-25 2016-04-20 엑시큐어, 인크. 예방 및 치료 용도를 위한 면역자극제로서의 구형 핵산-기재 구축물
RS59991B1 (sr) 2013-08-08 2020-04-30 Scripps Research Inst Metoda enzimskog in vitro obeležavanja specifičnog za položaj nukleinskih kiselina uvođenjem neprirodnih nukleotida
US10077444B2 (en) 2013-10-02 2018-09-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
TWI694080B (zh) 2013-10-04 2020-05-21 美國西奈山伊坎醫學院 抑制ａｌａｓ１基因表現的組合物及方法
WO2015054451A1 (en) 2013-10-09 2015-04-16 The United States Of America As Represented By The Secretary Department Of Health And Human Services Detection of hepatitis delta virus (hdv) for the diagnosis and treatment of sjögren's syndrome and lymphoma
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
WO2015061246A1 (en) 2013-10-21 2015-04-30 Isis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
EP3060680B1 (en) 2013-10-21 2019-02-27 The General Hospital Corporation Methods relating to circulating tumor cell clusters and the treatment of cancer
RU2694950C2 (ru) 2013-11-04 2019-07-18 Монсанто Текнолоджи Ллс Композиции и способы для борьбы с членистоногими паразитами и заражениями вредителями
US10301622B2 (en) 2013-11-04 2019-05-28 Northwestern University Quantification and spatio-temporal tracking of a target using a spherical nucleic acid (SNA)
EP3068440B1 (en) 2013-11-15 2020-01-08 Northwestern University Inhibition of oxidative stress in atrial fibrillation
CN105939699B (zh) 2013-12-03 2020-10-02 西北大学 脂质体颗粒、制备所述脂质体颗粒的方法以及其用途
CA2844640A1 (en) 2013-12-06 2015-06-06 The University Of British Columbia Method for treatment of castration-resistant prostate cancer
WO2015085183A2 (en) 2013-12-06 2015-06-11 Swift Biosciences, Inc. Cleavable competitor polynucleotides
EP3080269B1 (en) 2013-12-09 2019-05-22 Baylor College of Medicine Hippo and dystrophin complex signaling in cardiomyocyte renewal
UA119253C2 (uk) 2013-12-10 2019-05-27 Біолоджикс, Інк. Спосіб боротьби із вірусом у кліща varroa та у бджіл
BR112016013148B1 (pt) 2013-12-12 2024-02-27 Alnylam Pharmaceuticals, Inc ÁCIDO RIBONUCLEICO DE FITA DUPLA (dsRNA) PARA INIBIÇÃO DA EXPRESSÃO DO FATOR B DO COMPLEMENTO (CFB) EM UMA CÉLULA, COMPOSIÇÃO FARMACÊUTICA PARA INIBIÇÃO DA EXPRESSÃO DE UM GENE DO FATOR B DO COMPLEMENTO, SEU USO, E MÉTODO IN VITRO DE INIBIÇÃO DA EXPRESSÃO DO FATOR B DO COMPLEMENTO (CFB) EM UMA CÉLULA
AU2014364520B2 (en) 2013-12-20 2020-01-02 The General Hospital Corporation Methods and assays relating to circulating tumor cells
WO2015108982A2 (en) 2014-01-15 2015-07-23 Monsanto Technology Llc Methods and compositions for weed control using epsps polynucleotides
CA2937539A1 (en) 2014-02-04 2015-08-13 Genentech, Inc. Mutant smoothened and methods of using the same
CN106715695B (zh) 2014-02-05 2020-07-31 耶达研究及发展有限公司 用于治疗和诊断的微rna和包含所述微rna的组合物
EA201691587A1 (ru) 2014-02-11 2017-01-30 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ iRNA ДЛЯ КЕТОГЕКСОКИНАЗЫ (KHK) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
EP3119789B1 (en) 2014-03-17 2020-04-22 Ionis Pharmaceuticals, Inc. Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom
PL3119888T3 (pl) 2014-03-19 2021-12-20 Ionis Pharmaceuticals, Inc. Kompozycje do modulacji ekspresji ataksyny 2
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
SG11201608109TA (en) 2014-04-01 2016-10-28 Ionis Pharmaceuticals Inc Compositions for modulating sod-1 expression
CN106413390B (zh) 2014-04-01 2019-09-27 孟山都技术公司 用于控制虫害的组合物和方法
HUE056155T2 (hu) 2014-04-09 2022-01-28 Scripps Research Inst Nem-természetes vagy módosított nukleozid-trifoszfátok sejtekbe importálása nukleinsav-trifoszfát transzporterekkel
WO2015164693A1 (en) 2014-04-24 2015-10-29 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising alpha-beta-constrained nucleic acid
PH12016502062B1 (en) 2014-05-01 2023-01-27 Ionis Pharmaceuticals Inc Compositions and methods for modulating complement factor b expression
EP4223315A3 (en) 2014-05-01 2023-08-23 Ionis Pharmaceuticals, Inc. Method for synthesis of reactive conjugate clusters
TW201607559A (zh) 2014-05-12 2016-03-01 阿尼拉製藥公司 治療ｓｅｒｐｉｎｃ１相關疾患之方法和組成物
EA201692370A1 (ru) 2014-05-22 2017-03-31 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ иРНК АНГИОТЕНЗИНОГЕНА (AGT) И СПОСОБЫ ИХ ИСПОЛЬЗОВАНИЯ
CA2950589A1 (en) 2014-06-02 2015-12-10 Children's Medical Center Corporation Methods and compositions for immunomodulation
US10434064B2 (en) 2014-06-04 2019-10-08 Exicure, Inc. Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
WO2015190922A1 (en) 2014-06-10 2015-12-17 Erasmus University Medical Center Rotterdam Antisense oligonucleotides useful in treatment of pompe disease
CA2953347A1 (en) 2014-06-23 2015-12-30 Monsanto Technology Llc Compositions and methods for regulating gene expression via rna interference
US11807857B2 (en) 2014-06-25 2023-11-07 Monsanto Technology Llc Methods and compositions for delivering nucleic acids to plant cells and regulating gene expression
EP3760208B1 (en) 2014-06-25 2024-05-29 The General Hospital Corporation Targeting human satellite ii (hsatii)
ES2799404T3 (es) 2014-07-15 2020-12-17 Yissum Research And Development Company Of The Hebrew Univ Of Jerusalem Ltd Polipéptidos de CD44 aislados y usos de los mismos
US9951327B1 (en) 2014-07-17 2018-04-24 Integrated Dna Technologies, Inc. Efficient and rapid method for assembling and cloning double-stranded DNA fragments
RU2021123470A (ru) 2014-07-29 2021-09-06 Монсанто Текнолоджи Ллс Композиции и способы борьбы с насекомыми-вредителями
EP3189069B1 (en) 2014-07-31 2024-10-23 UAB Research Foundation Apoe mimetic peptides and higher potency to clear plasma cholesterol
CN106794256B (zh) 2014-08-19 2021-04-30 西北大学 蛋白质/寡核苷酸核-壳纳米颗粒治疗剂
WO2016030899A1 (en) 2014-08-28 2016-03-03 Yeda Research And Development Co. Ltd. Methods of treating amyotrophic lateral scleroses
DK3185957T3 (da) 2014-08-29 2022-08-29 Alnylam Pharmaceuticals Inc Patisiran til anvendelse ved behandlingen af transthyretin-medieret amyloidosis
AU2015308721B2 (en) 2014-08-29 2021-04-01 Children's Medical Center Corporation Methods and compositions for the treatment of cancer
WO2016033424A1 (en) 2014-08-29 2016-03-03 Genzyme Corporation Methods for the prevention and treatment of major adverse cardiovascular events using compounds that modulate apolipoprotein b
WO2016040589A1 (en) 2014-09-12 2016-03-17 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting complement component c5 and methods of use thereof
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
EP3198012B1 (en) 2014-09-26 2019-09-04 University of Massachusetts Rna-modulating agents
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
EP3207138B1 (en) 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3904519A1 (en) 2014-10-30 2021-11-03 Genzyme Corporation Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
AU2015350120B2 (en) 2014-11-17 2021-05-27 Alnylam Pharmaceuticals, Inc. Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof
EP3220895B1 (en) 2014-11-21 2022-08-31 Northwestern University The sequence-specific cellular uptake of spherical nucleic acid nanoparticle conjugates
CN107532162A (zh) 2014-12-12 2018-01-02 托德·M·伍尔夫 用于利用寡核苷酸编辑细胞中核酸的组合物和方法
US9688707B2 (en) 2014-12-30 2017-06-27 Ionis Pharmaceuticals, Inc. Bicyclic morpholino compounds and oligomeric compounds prepared therefrom
WO2016112132A1 (en) 2015-01-06 2016-07-14 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
US10538763B2 (en) 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
JP6942632B2 (ja) 2015-01-22 2021-09-29 モンサント テクノロジー エルエルシー Ｌｅｐｔｉｎｏｔａｒｓａ防除用組成物及びその方法
EP3247988A4 (en) 2015-01-23 2018-12-19 Vanderbilt University A robust interferometer and methods of using same
JP6929791B2 (ja) 2015-02-09 2021-09-01 デューク ユニバーシティ エピゲノム編集のための組成物および方法
CA2976445A1 (en) 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
US11421229B2 (en) 2015-02-20 2022-08-23 Baylor College Of Medicine p63 inactivation for the treatment of heart failure
EP3262181B1 (en) 2015-02-23 2024-04-10 Ionis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
WO2016135559A2 (en) 2015-02-23 2016-09-01 Crispr Therapeutics Ag Materials and methods for treatment of human genetic diseases including hemoglobinopathies
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
WO2016142948A1 (en) 2015-03-11 2016-09-15 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Decoy oligonucleotides for the treatment of diseases
WO2016154629A1 (en) 2015-03-26 2016-09-29 Woman & Infants' Hospital Of Rhode Island Therapy for malignant disease
BR112017020750A2 (pt) 2015-03-27 2018-06-26 Harvard College células t modificadas e métodos de produção e utilização das mesmas
EP3273971A4 (en) 2015-03-27 2018-12-05 Yeda Research and Development Co. Ltd. Methods of treating motor neuron diseases
WO2016164463A1 (en) 2015-04-07 2016-10-13 The General Hospital Corporation Methods for reactivating genes on the inactive x chromosome
MX2017012610A (es) 2015-04-08 2018-03-16 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion del gen lect2.
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
US10907152B2 (en) 2015-05-04 2021-02-02 Monsanto Technology Llc Compositions and methods for controlling arthropod parasite and pest infestations
HK1251010A1 (zh) 2015-05-06 2019-01-18 Benitec IP Holdings Inc. 用於治疗乙型肝炎病毒(hbv)感染的试剂及用途
EP3294280A1 (en) 2015-05-11 2018-03-21 Yeda Research and Development Co., Ltd. Citrin inhibitors for the treatment of cancer
UY36703A (es) 2015-06-02 2016-12-30 Monsanto Technology Llc Composiciones y métodos para la administración de un polinucleótido en una planta
WO2016196782A1 (en) 2015-06-03 2016-12-08 Monsanto Technology Llc Methods and compositions for introducing nucleic acids into plants
EP3307316A1 (en) 2015-06-12 2018-04-18 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
WO2016205323A1 (en) 2015-06-18 2016-12-22 Alnylam Pharmaceuticals, Inc. Polynucleotde agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
US20180188257A1 (en) 2015-06-19 2018-07-05 University Of Rochester Septin proteins as novel biomarkers for detection and treatment of müllerian cancers
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
CN108139375A (zh) 2015-06-26 2018-06-08 贝斯以色列女执事医疗中心股份有限公司 靶向髓样衍生的抑制细胞中的四跨膜蛋白33(tspan33)的癌症疗法
EP3314027A4 (en) 2015-06-29 2019-07-03 Caris Science, Inc. THERAPEUTIC OLIGONUCLEOTIDES
WO2017011286A1 (en) 2015-07-10 2017-01-19 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (igfals) and insulin-like growth factor 1 (igf-1) irna compositions and methods of use thereof
EP3328873B1 (en) 2015-07-28 2025-09-17 Caris Science, Inc. Targeted oligonucleotides
US20180221393A1 (en) 2015-08-03 2018-08-09 Biokine Therapeutics Ltd. Cxcr4 binding agents for treatment of diseases
EP3332009A1 (en) 2015-08-04 2018-06-13 Yeda Research and Development Co., Ltd. Methods of screening for riboswitches and attenuators
EA201890619A1 (ru) 2015-09-02 2018-09-28 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ iRNA ЛИГАНДА 1 БЕЛКА ЗАПРОГРАММИРОВАННОЙ ГИБЕЛИ КЛЕТОК 1 (PD-L1) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
US20190048340A1 (en) 2015-09-24 2019-02-14 Crispr Therapeutics Ag Novel family of rna-programmable endonucleases and their uses in genome editing and other applications
EP3353303B1 (en) 2015-09-25 2023-08-02 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
WO2017066497A2 (en) 2015-10-13 2017-04-20 Duke University Genome engineering with type i crispr systems in eukaryotic cells
EP3368063B1 (en) 2015-10-28 2023-09-06 Vertex Pharmaceuticals Inc. Materials and methods for treatment of duchenne muscular dystrophy
PL3368578T3 (pl) 2015-10-30 2021-07-26 F. Hoffmann-La Roche Ag Przeciwciała anty-Htra1 i sposoby ich stosowania
WO2017075670A1 (en) 2015-11-05 2017-05-11 Children's Hospital Los Angeles "mobilizing leukemia cells"
US11866727B2 (en) 2015-11-06 2024-01-09 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1A
WO2017081686A1 (en) 2015-11-10 2017-05-18 B. G. Negev Technologies And Applications Ltd., At Ben-Gurion University Means and methods for reducing tumorigenicity of cancer stem cells
AU2016355178B9 (en) 2015-11-19 2019-05-30 Massachusetts Institute Of Technology Lymphocyte antigen CD5-like (CD5L)-interleukin 12B (p40) heterodimers in immunity
ES2930643T3 (es) 2015-11-23 2022-12-20 Univ California Seguimiento y manipulación de ARN celular a través de la administración nuclear de CRISPR/CAS9
WO2017095967A2 (en) 2015-11-30 2017-06-08 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
US11851653B2 (en) 2015-12-01 2023-12-26 Crispr Therapeutics Ag Materials and methods for treatment of alpha-1 antitrypsin deficiency
WO2017096395A1 (en) 2015-12-04 2017-06-08 Ionis Pharmaceuticals, Inc. Methods of treating breast cancer
JP6985283B2 (ja) 2015-12-07 2021-12-22 ジェンザイム・コーポレーション Ｓｅｒｐｉｎｃ１関連障害を処置するための方法および組成物
AU2015416656B2 (en) 2015-12-07 2023-02-23 Erasmus University Medical Center Rotterdam Enzymatic replacement therapy and antisense therapy for Pompe disease
WO2017106767A1 (en) 2015-12-18 2017-06-22 The Scripps Research Institute Production of unnatural nucleotides using a crispr/cas9 system
MX2018007840A (es) 2015-12-23 2019-05-02 Crispr Therapeutics Ag Materiales y metodos para el tratamiento de la esclerosis lateral amiotrofica y/o la degeneracion lobar frontotemporal.
AU2016381174A1 (en) 2015-12-31 2018-05-31 Ionis Pharmaceuticals, Inc. Methods for reducing Ataxin-2 expression
WO2017120365A1 (en) 2016-01-05 2017-07-13 Ionis Pharmaceuticals, Inc. Methods for reducing lrrk2 expression
WO2017132483A1 (en) 2016-01-29 2017-08-03 Vanderbilt University Free-solution response function interferometry
EP3411078A1 (en) 2016-02-02 2018-12-12 Crispr Therapeutics AG Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
WO2017136558A1 (en) 2016-02-04 2017-08-10 Curis, Inc. Mutant smoothened and methods of using the same
WO2017141109A1 (en) 2016-02-18 2017-08-24 Crispr Therapeutics Ag Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
JP7033072B2 (ja) 2016-02-25 2022-03-09 ザ ブリガム アンド ウィメンズ ホスピタル インコーポレイテッド Ｓｍｏｃ２を標的化する線維症のための治療方法
CA3016592A1 (en) 2016-03-04 2017-09-08 Rhode Island Hospital Targeting microrna for cancer treatment
US10577607B2 (en) 2016-03-16 2020-03-03 Ionis Pharmaceuticals, Inc. Modulation of DYRK1B expression
US11083799B2 (en) 2016-03-16 2021-08-10 Crispr Therapeutics Ag Materials and methods for treatment of hereditary haemochromatosis
EP3429690A4 (en) 2016-03-16 2019-10-23 Ionis Pharmaceuticals, Inc. METHOD FOR MODULATING KEAP1
US10731166B2 (en) 2016-03-18 2020-08-04 Caris Science, Inc. Oligonucleotide probes and uses thereof
US20190127713A1 (en) 2016-04-13 2019-05-02 Duke University Crispr/cas9-based repressors for silencing gene targets in vivo and methods of use
CA3020754C (en) 2016-04-14 2023-07-25 Benitec Biopharma Limited Reagents for treatment of oculopharyngeal muscular dystrophy (opmd) and use thereof
MX2018012729A (es) 2016-04-18 2019-07-04 Crispr Therapeutics Ag Materiales y metodos para el tratamiento de hemoglobinopatias.
MA45295A (fr) 2016-04-19 2019-02-27 Alnylam Pharmaceuticals Inc Composition d'arni de protéine de liaison de lipoprotéines haute densité (hdlbp/vigiline) et procédés pour les utiliser
WO2017191503A1 (en) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
CA3025486A1 (en) 2016-05-25 2017-11-30 Caris Science, Inc. Oligonucleotide probes and uses thereof
EP3469083A1 (en) 2016-06-10 2019-04-17 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
US11236339B2 (en) 2016-06-17 2022-02-01 Ionis Pharmaceuticals, Inc. Modulation of GYS1 expression
DK3475295T3 (da) 2016-06-24 2022-10-24 Scripps Research Inst Hidtil ukendt nukleosidtriphosphat-transportør og anvendelser deraf
EP3478313B1 (en) 2016-06-29 2022-05-04 CRISPR Therapeutics AG Materials and methods for treatment of amyotrophic lateral sclerosis (als) and other related disorders
WO2018002812A1 (en) 2016-06-29 2018-01-04 Crispr Therapeutics Ag Materials and methods for treatment of myotonic dystrophy type 1 (dm1) and other related disorders
US11564997B2 (en) 2016-06-29 2023-01-31 Crispr Therapeutics Ag Materials and methods for treatment of friedreich ataxia and other related disorders
US11459587B2 (en) 2016-07-06 2022-10-04 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
CA3029132A1 (en) 2016-07-06 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of pain related disorders
WO2018007871A1 (en) 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
AU2017296195A1 (en) 2016-07-11 2019-01-24 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
EP4275747A3 (en) 2016-07-19 2024-01-24 Duke University Therapeutic applications of cpf1-based genome editing
KR20190031306A (ko) 2016-07-21 2019-03-25 맥스시티 인코포레이티드 게놈 dna를 변경하기 위한 방법 및 조성물
WO2018020323A2 (en) 2016-07-25 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of fatty acid disorders
NL2017295B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Antisense oligomeric compound for Pompe disease
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
US11364304B2 (en) 2016-08-25 2022-06-21 Northwestern University Crosslinked micellar spherical nucleic acids
WO2018045317A1 (en) 2016-09-02 2018-03-08 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
CN109661233A (zh) 2016-10-06 2019-04-19 Ionis 制药公司 缀合低聚化合物的方法
EP3532638A4 (en) 2016-10-31 2020-07-29 University of Massachusetts TARGETING MICROARN-101-3 P IN CARCINOTHERAPY
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
TWI788312B (zh) 2016-11-23 2023-01-01 美商阿尼拉製藥公司 絲胺酸蛋白酶抑制因子Ａ１ ｉＲＮＡ組成物及其使用方法
EP3330276A1 (en) 2016-11-30 2018-06-06 Universität Bern Novel bicyclic nucleosides and oligomers prepared therefrom
US11033570B2 (en) 2016-12-02 2021-06-15 Cold Spring Harbor Laboratory Modulation of Lnc05 expression
JP7206214B2 (ja) 2016-12-13 2023-01-17 シアトル チルドレンズ ホスピタル （ディービーエイ シアトル チルドレンズ リサーチ インスティテュート） インビトロ及びインビボで操作された細胞において発現された化学誘導シグナル伝達複合体の外因性薬物活性化の方法
EP3555292A1 (en) 2016-12-16 2019-10-23 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing ttr-associated diseases using transthyretin (ttr) irna compositions
SG11201906735RA (en) 2017-01-23 2019-08-27 Regeneron Pharma Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
US20200216857A1 (en) 2017-02-22 2020-07-09 Crispr Therapeutics Ag Materials and methods for treatment of spinocerebellar ataxia type 2 (sca2) and other spinocerebellar ataxia type 2 protein (atxn2) gene related conditions or disorders
EP3585807A1 (en) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of early onset parkinson's disease (park1) and other synuclein, alpha (snca) gene related conditions or disorders
EP3585898A1 (en) 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of spinocerebellar ataxia type 1 (sca1) and other spinocerebellar ataxia type 1 protein (atxn1) gene related conditions or disorders
US11407997B2 (en) 2017-02-22 2022-08-09 Crispr Therapeutics Ag Materials and methods for treatment of primary hyperoxaluria type 1 (PH1) and other alanine-glyoxylate aminotransferase (AGXT) gene related conditions or disorders
CN110662838B (zh) 2017-02-22 2024-05-28 克里斯珀医疗股份公司 用于基因编辑的组合物和方法
WO2018165564A1 (en) 2017-03-09 2018-09-13 Ionis Pharmaceuticals, Inc. Morpholino modified oligomeric compounds
US20180284123A1 (en) 2017-03-30 2018-10-04 California Institute Of Technology Barcoded rapid assay platform useful for efficient analysis of candidate molecules and methods of making and using the platform
CA3059446A1 (en) 2017-04-18 2018-10-25 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (hbv) infection
WO2018195338A1 (en) 2017-04-20 2018-10-25 Atyr Pharma, Inc. Compositions and methods for treating lung inflammation
EP4036101A1 (en) 2017-04-20 2022-08-03 Synthena AG Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
WO2018193428A1 (en) 2017-04-20 2018-10-25 Synthena Ag Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
WO2018195486A1 (en) 2017-04-21 2018-10-25 The Broad Institute, Inc. Targeted delivery to beta cells
WO2018208998A1 (en) 2017-05-10 2018-11-15 The Regents Of The University Of California Directed editing of cellular rna via nuclear delivery of crispr/cas9
EP3621981A2 (en) 2017-05-12 2020-03-18 CRISPR Therapeutics AG Materials and methods for engineering cells and uses thereof in immuno-oncology
TW202434265A (zh) 2017-07-10 2024-09-01 美商健贊公司 於患有血友病之個體中治療出血事件之方法及組成物
IL271903B2 (en) 2017-07-11 2025-04-01 Synthorx Inc Combination of unnatural nucleotides and methods therefor
MA49716A (fr) 2017-07-11 2021-04-07 Scripps Research Inst Incorporation de nucléotides non naturels et procédés d'utilisationin vivo
KR20200028997A (ko) 2017-07-13 2020-03-17 노오쓰웨스턴 유니버시티 올리고뉴클레오타이드-작용화된 금속-유기 프레임워크 나노입자를 제조하는 일반적이고 직접적인 방법
CA3069868A1 (en) 2017-07-13 2019-01-17 Alnylam Pharmaceuticals Inc. Lactate dehydrogenase a (ldha) irna compositions and methods of use thereof
SG11202000939PA (en) 2017-08-03 2020-02-27 Synthorx Inc Cytokine conjugates for the treatment of proliferative and infectious diseases
WO2019036613A1 (en) 2017-08-18 2019-02-21 Ionis Pharmaceuticals, Inc. MODULATION OF THE NOTCH SIGNALING PATHWAY FOR THE TREATMENT OF RESPIRATORY DISORDERS
US10517889B2 (en) 2017-09-08 2019-12-31 Ionis Pharmaceuticals, Inc. Modulators of SMAD7 expression
WO2019055460A1 (en) 2017-09-13 2019-03-21 The Children's Medical Center Corporation COMPOSITIONS AND METHODS FOR THE TREATMENT OF TRANSPOSON-ASSOCIATED DISEASES
US11806360B2 (en) 2017-09-19 2023-11-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (TTR) mediated amyloidosis
EP3697907A1 (en) 2017-10-17 2020-08-26 CRISPR Therapeutics AG Compositions and methods for gene editing for hemophilia a
MA50849A (fr) 2017-10-26 2020-09-02 Vertex Pharma Substances et procédés pour le traitement d'hémoglobinopathies
CA3078971A1 (en) 2017-11-01 2019-05-09 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
US20210032622A1 (en) 2017-11-09 2021-02-04 Crispr Therapeutics Ag Self-inactivating (sin) crispr/cas or crispr/cpf1 systems and uses thereof
EP3710587A1 (en) 2017-11-16 2020-09-23 Alnylam Pharmaceuticals, Inc. Kisspeptin 1 (kiss1) irna compositions and methods of use thereof
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
MA50877A (fr) 2017-11-21 2020-09-30 Bayer Healthcare Llc Matériaux et méthodes pour le traitement de la rétinite pigmentaire autosomique dominante
WO2019104155A2 (en) 2017-11-22 2019-05-31 The University Of Chicago Chemical probe-dependent evaluation of protein activity and uses thereof
US12161674B2 (en) 2017-12-05 2024-12-10 Vertex Pharmaceuticals Incorporated CRISPR-CAS9 modified CD34+ human hematopoietic stem and progenitor cells and uses thereof
AU2018386301A1 (en) 2017-12-14 2020-06-18 Bayer Healthcare Llc Novel RNA-programmable endonuclease systems and their use in genome editing and other applications
CN111727252A (zh) 2017-12-18 2020-09-29 阿尔尼拉姆医药品有限公司 高速泳动族盒-1（HMGB1）iRNA组合物及其使用方法
AU2018393050A1 (en) 2017-12-21 2020-06-18 Bayer Healthcare Llc Materials and methods for treatment of Usher Syndrome Type 2A
WO2019126641A2 (en) 2017-12-21 2019-06-27 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
CA3084633A1 (en) 2017-12-21 2019-06-27 Crispr Therapeutics Ag Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
CA3087234A1 (en) 2017-12-29 2019-07-04 The Scripps Research Institute Unnatural base pair compositions and methods of use
EP3737424A4 (en) 2018-01-10 2021-10-27 Translate Bio MA, Inc. COMPOSITIONS AND METHODS TO FACILITATE THE DELIVERY OF SYNTHETIC NUCLEIC ACIDS INTO CELLS
EP3737762A1 (en) 2018-01-12 2020-11-18 CRISPR Therapeutics AG Compositions and methods for gene editing by targeting transferrin
CA3088522A1 (en) 2018-01-15 2019-07-18 Ionis Pharmaceuticals, Inc. Modulators of dnm2 expression
US12319667B2 (en) 2018-01-19 2025-06-03 Synthena Ag Tricyclo-DNA nucleoside precursors and processes for preparing the same
WO2019147743A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
EP3749768A1 (en) 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
US11566236B2 (en) 2018-02-05 2023-01-31 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
EP3749368A1 (en) 2018-02-08 2020-12-16 Yeda Research and Development Co. Ltd Methods of identifying and using agents for treating diseases associated with intestinal barrier dysfunction
MA51869A (fr) 2018-02-16 2020-12-23 Bayer Healthcare Llc Compositions et méthodes pour l'édition génique par ciblage du fibrinogène-alpha
WO2019165453A1 (en) 2018-02-26 2019-08-29 Synthorx, Inc. Il-15 conjugates and uses thereof
TWI840345B (zh) 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
WO2019169243A1 (en) 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-beta precursor protein
US11897911B2 (en) 2018-03-07 2024-02-13 Sanofi Nucleotide precursors, nucleotide analogs and oligomeric compounds containing the same
MA52074A (fr) 2018-03-19 2021-01-27 Bayer Healthcare Llc Nouveaux systèmes d'endonucléase à arn programmable et leurs utilisations
US11661601B2 (en) 2018-03-22 2023-05-30 Ionis Pharmaceuticals, Inc. Methods for modulating FMR1 expression
WO2019186514A2 (en) 2018-03-30 2019-10-03 Rheinische Friedrich-Wilhelms-Universitat Bonn Aptamers for targeted activaton of t cell-mediated immunity
US20210155959A1 (en) 2018-04-06 2021-05-27 Children's Medical Center Corporation Compositions and methods for somatic cell reprogramming and modulating imprinting
PE20201349A1 (es) 2018-04-11 2020-11-30 Ionis Pharmaceuticals Inc Moduladores de la expresion de ezh2
WO2019204668A1 (en) 2018-04-18 2019-10-24 Casebia Therapeutics Limited Liability Partnership Compositions and methods for knockdown of apo(a) by gene editing for treatment of cardiovascular disease
SG11202007874XA (en) 2018-04-27 2020-09-29 Seattle Childrens Hospital Dba Seattle Childrens Res Inst Rapamycin resistant cells
CU20200082A7 (es) 2018-05-09 2021-06-08 Ionis Pharmaceuticals Inc Compuestos y métodos para la reducción de la expresión de fxi
UY38225A (es) 2018-05-09 2019-11-29 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir de la expresión de atxn3
TWI851574B (zh) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原（ＡＧＴ）ｉＲＮＡ組成物及其使用方法
WO2019241648A1 (en) 2018-06-14 2019-12-19 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing stmn2 expression
TWI833770B (zh) 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
CN112585268B (zh) 2018-06-28 2025-03-04 克里斯珀医疗股份公司 通过插入供体多核苷酸用于基因组编辑的组合物和方法
US12496311B2 (en) 2018-07-17 2025-12-16 Aronora, Inc. Methods for safely reducing thrombopoietin
EP3826645A4 (en) 2018-07-25 2023-05-17 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING ATXN2 EXPRESSION
BR112021001613A2 (pt) 2018-08-13 2021-05-04 Alnylam Pharmaceuticals, Inc. agentes de ácido ribonucleico de fita dupla, célula, composições farmacêuticas, métodos de inibição da expressão gênica, de inibição da replicação e de tratar um sujeito, métodos para reduzir o nível de um antígeno e para reduzir a carga viral e uso de um agente de dsrna
WO2020037125A1 (en) 2018-08-16 2020-02-20 Alnylam Pharmaceuticals Inc. Compositions and methods for inhibiting expression of the lect2 gene
CA3110661A1 (en) 2018-08-29 2020-03-05 University Of Massachusetts Inhibition of protein kinases to treat friedreich ataxia
EP3849584A4 (en) 2018-09-14 2022-06-22 Northwestern University PROGRAMMING PROTEIN POLYMERIZATION WITH DNA
US20210332367A1 (en) 2018-09-18 2021-10-28 Alnylam Pharmaceuticals, Inc. KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP3856931B1 (en) 2018-09-25 2023-10-11 Co-Diagnostics, Inc. Allele-specific design of cooperative primers for improved nucleic acid variant genotyping
MX2021004455A (es) 2018-10-17 2021-08-11 Crispr Therapeutics Ag Composiciones y métodos para administrar transgenes.
US10913951B2 (en) 2018-10-31 2021-02-09 University of Pittsburgh—of the Commonwealth System of Higher Education Silencing of HNF4A-P2 isoforms with siRNA to improve hepatocyte function in liver failure
EP3876973B1 (en) 2018-11-08 2025-10-22 Synthorx, Inc. Interleukin 10 conjugates and uses thereof
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
EP3880821A4 (en) 2018-11-15 2023-01-25 Ionis Pharmaceuticals, Inc. IRF5 EXPRESSION MODULATORS
TWI841633B (zh) 2018-11-21 2024-05-11 美商Ionis製藥公司 用於減少朊病毒表現之化合物及方法
IL263184A (en) 2018-11-21 2020-05-31 Yarden Yosef Method of treating cancer and compositions for same
AU2019387219B2 (en) 2018-11-28 2025-04-24 Bayer Healthcare Llc Optimized mRNA encoding CAS9 for use in LNPs
US12485136B2 (en) 2018-12-03 2025-12-02 Takeda Pharmaceuticals U.S.A., Inc. Methods for the treatment of trinucleotide repeat expansion disorders associated with MLH3 activity
US20210332495A1 (en) 2018-12-06 2021-10-28 Northwestern University Protein Crystal Engineering Through DNA Hybridization Interactions
CA3123617A1 (en) 2018-12-20 2020-06-25 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of kcnt1 related disorders
HRP20231338T1 (hr) 2018-12-20 2024-02-16 Vir Biotechnology, Inc. Kombinirana terapija hbv
MX2021008628A (es) 2019-01-16 2021-11-17 Genzyme Corp Composiciones de arni para serpinc1 y metodos de uso de las mismas.
EP3917540A1 (en) 2019-01-31 2021-12-08 Ionis Pharmaceuticals, Inc. Modulators of yap1 expression
EP3923974A4 (en) 2019-02-06 2023-02-08 Synthorx, Inc. IL-2 CONJUGATES AND METHODS OF USE THEREOF
MA54951A (fr) 2019-02-15 2021-12-22 Bayer Healthcare Llc Édition de gène pour l'hémophilie a avec une expression de facteur viii améliorée
WO2020171889A1 (en) 2019-02-19 2020-08-27 University Of Rochester Blocking lipid accumulation or inflammation in thyroid eye disease
EP3927378A1 (en) 2019-02-21 2021-12-29 Yissum Research Development Company of the Hebrew University of Jerusalem Ltd. Method for reduction drug-induced nephrotoxicity
KR20210134686A (ko) 2019-02-27 2021-11-10 아이오니스 파마수티컬즈, 인코포레이티드 Malat1 발현의 조절인자
SG11202109741VA (en) 2019-03-12 2021-10-28 Crispr Therapeutics Ag Novel high fidelity rna-programmable endonuclease systems and uses thereof
AU2020253823A1 (en) 2019-03-29 2021-10-14 Dicerna Pharmaceuticals, Inc. Compositions and methods for the treatment of KRAS associated diseases or disorders
TWI874376B (zh) 2019-03-29 2025-03-01 美商Ionis製藥公司 用於調節ube3a-ats 之化合物及方法
CN114174532A (zh) 2019-04-05 2022-03-11 德克萨斯大学系统董事会 细胞条形码编码的方法和应用
CA3136676A1 (en) 2019-05-03 2020-11-12 Dicerna Pharmaceuticals, Inc. Double-stranded nucleic acid inhibitor molecules with shortened sense strands
EP3966327A1 (en) 2019-05-08 2022-03-16 Vertex Pharmaceuticals Incorporated Crispr/cas all-in-two vector systems for treatment of dmd
KR20220036914A (ko) 2019-05-13 2022-03-23 비르 바이오테크놀로지, 인코포레이티드 B형 간염 바이러스(hbv) 감염을 치료하기 위한 조성물 및 방법
AU2020291535A1 (en) 2019-06-14 2022-01-20 The Scripps Research Institute Reagents and methods for replication, transcription, and translation in semi-synthetic organisms
WO2020257325A1 (en) 2019-06-17 2020-12-24 Vertex Pharmaceuticals Inc. Compositions and methods for editing beta-globin for treatment of hemaglobinopathies
EP3956450B1 (en) 2019-07-26 2025-08-13 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gfap
EP4007811A2 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Carboxypeptidase b2 (cpb2) irna compositions and methods of use thereof
EP4007812A1 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof
EP4013870A1 (en) 2019-08-13 2022-06-22 Alnylam Pharmaceuticals, Inc. Small ribosomal protein subunit 25 (rps25) irna agent compositions and methods of use thereof
TWI873169B (zh) 2019-08-15 2025-02-21 美商欣爍克斯公司 使用il-2接合物之免疫腫瘤學組合療法
JP2022544587A (ja) 2019-08-15 2022-10-19 アイオーニス ファーマシューティカルズ， インコーポレーテッド 結合修飾オリゴマー化合物及びその使用
BR112022003046A8 (pt) 2019-08-23 2024-02-06 Synthorx Inc Conjugado de il-15, seu método de preparo e seus usos, bem como composição farmacêutica e método in vitro de expansão de uma ou mais das populações de células t
MX2022002689A (es) 2019-09-03 2022-04-07 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion del gen de quimiotaxina derivada de celulas de leucocitos 2 (lect2).
TW202124385A (zh) 2019-09-10 2021-07-01 美商欣爍克斯公司 治療自體免疫疾病之il-2接合物及使用方法
US12319711B2 (en) 2019-09-20 2025-06-03 Northwestern University Spherical nucleic acids with tailored and active protein coronae
US12503699B2 (en) 2019-10-04 2025-12-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing UGT1a1 gene expression
WO2021076828A1 (en) 2019-10-18 2021-04-22 Alnylam Pharmaceuticals, Inc. Solute carrier family member irna compositions and methods of use thereof
KR20220084399A (ko) 2019-10-22 2022-06-21 알닐람 파마슈티칼스 인코포레이티드 보체 성분 C3 iRNA 조성물 및 이의 사용 방법
US12378560B2 (en) 2019-10-29 2025-08-05 Northwestern University Sequence multiplicity within spherical nucleic acids
EP4051796A1 (en) 2019-11-01 2022-09-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
JP2023500661A (ja) 2019-11-01 2023-01-10 アルナイラム ファーマシューティカルズ， インコーポレイテッド ハンチンチン（ＨＴＴ）ｉＲＮＡ剤組成物およびその使用方法
CA3156405A1 (en) 2019-11-04 2021-05-14 Synthorx, Inc. Interleukin 10 conjugates and uses thereof
WO2021096763A1 (en) 2019-11-13 2021-05-20 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
WO2021102373A1 (en) 2019-11-22 2021-05-27 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3) rnai agent compositions and methods of use thereof
CN115335521A (zh) 2019-11-27 2022-11-11 克里斯珀医疗股份公司 合成rna分子的方法
CN115151641A (zh) 2019-12-13 2022-10-04 阿尔尼拉姆医药品有限公司 人类染色体9开放阅读框72(C9ORF72)iRNA剂组合物及其使用方法
WO2021126734A1 (en) 2019-12-16 2021-06-24 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
WO2021122944A1 (en) 2019-12-18 2021-06-24 Alia Therapeutics Srl Compositions and methods for treating retinitis pigmentosa
BR112022013821A2 (pt) 2020-01-15 2022-09-13 Dicerna Pharmaceuticals Inc Ácidos nucleicos de 4?-o-metileno fosfonato e seus análogos
WO2021154705A1 (en) 2020-01-27 2021-08-05 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Rab13 and net1 antisense oligonucleotides to treat metastatic cancer
WO2021154941A1 (en) 2020-01-31 2021-08-05 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions for use in the treatment of amyotrophic lateral sclerosis (als)
WO2021163066A1 (en) 2020-02-10 2021-08-19 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing vegf-a expression
JP7735288B2 (ja) 2020-02-18 2025-09-08 アルナイラム ファーマシューティカルズ， インコーポレイテッド アポリポタンパク質Ｃ３（ＡＰＯＣ３）ｉＲＮＡ組成物およびその使用法
BR112022016238A2 (pt) 2020-02-28 2022-10-11 Ionis Pharmaceuticals Inc Compostos e métodos para modular smn2
WO2021178607A1 (en) 2020-03-05 2021-09-10 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
BR112022017822A2 (pt) 2020-03-06 2022-11-08 Alnylam Pharmaceuticals Inc Composições de irna de cetoexocinase (khk) e métodos de uso das mesmas
EP4121534A1 (en) 2020-03-18 2023-01-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
TW202204615A (zh) 2020-03-26 2022-02-01 美商阿尼拉製藥公司 冠狀病毒ｉＲＮＡ組成物及其使用方法
EP4127171A2 (en) 2020-03-30 2023-02-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
WO2021207167A1 (en) 2020-04-06 2021-10-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
WO2021206917A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2021206922A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
WO2021207189A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
CN115955972A (zh) 2020-04-27 2023-04-11 阿尔尼拉姆医药品有限公司 载脂蛋白E(APOE)iRNA剂组合物及其使用方法
JP2023523790A (ja) 2020-04-30 2023-06-07 アルナイラム ファーマシューティカルズ， インコーポレイテッド 補体因子Ｂ（ＣＦＢ）ｉＲＮＡ組成物およびその使用方法
CA3181546A1 (en) 2020-05-01 2021-11-04 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating atxn1
WO2021231692A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
EP4150077A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
WO2021231679A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
WO2021231680A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
EP4150089A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1)
WO2021231673A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
CA3162416C (en) 2020-05-15 2023-07-04 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
EP4150078A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
US20230183707A1 (en) 2020-05-21 2023-06-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
EP4153747A2 (en) 2020-05-22 2023-03-29 Wave Life Sciences Ltd. Double stranded oligonucleotide compositions and methods relating thereto
AR122534A1 (es) 2020-06-03 2022-09-21 Triplet Therapeutics Inc Métodos para el tratamiento de los trastornos de expansión por repetición de nucleótidos asociados con la actividad de msh3
WO2021248052A1 (en) 2020-06-05 2021-12-09 The Broad Institute, Inc. Compositions and methods for treating neoplasia
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
EP4649951A2 (en) 2020-06-09 2025-11-19 Alnylam Pharmaceuticals, Inc. Sirna compositions and methods for silencing gpam (glycerol-3-phosphate acyltransferase 1, mitochondrial) expression
CN116209760A (zh) 2020-06-18 2023-06-02 阿尔尼拉姆医药品有限公司 黄嘌呤脱氢酶(XDH)iRNA组合物及其使用方法
CN116390943A (zh) 2020-06-24 2023-07-04 维尔生物科技有限公司 工程化乙型肝炎病毒中和抗体和其用途
JP2023531509A (ja) 2020-06-25 2023-07-24 シンソークス， インコーポレイテッド Ｉｌ－２コンジュゲートおよび抗ｅｇｆｒ抗体を用いる免疫腫瘍学併用療法
TW202216996A (zh) 2020-06-29 2022-05-01 美商Ionis製藥公司 調節ｐｌｐ１之化合物及方法
UY39344A (es) 2020-07-28 2022-02-25 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de app
US20230277675A1 (en) 2020-08-04 2023-09-07 Dicerna Pharmaceuticals, Inc. Systemic delivery of oligonucleotides
CA3186935A1 (en) 2020-08-07 2022-02-10 Paymaan JAFAR-NEJAD Compounds and methods for modulating scn2a
EP4217489A1 (en) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidyl peptidase 4 (dpp4) irna compositions and methods of use thereof
WO2022070107A1 (en) 2020-09-30 2022-04-07 Crispr Therapeutics Ag Materials and methods for treatment of amyotrophic lateral sclerosis
EP3978608A1 (en) 2020-10-05 2022-04-06 SQY Therapeutics Oligomeric compound for dystrophin rescue in dmd patients throughout skipping of exon-51
JP2023544413A (ja) 2020-10-05 2023-10-23 アルナイラム ファーマシューティカルズ， インコーポレイテッド Ｇタンパク質共役受容体７５（ＧＰＲ７５）ｉＲＮＡ組成物およびその使用方法
WO2022076859A1 (en) 2020-10-09 2022-04-14 Synthorx, Inc. Immuno oncology therapies with il-2 conjugates
MX2023004029A (es) 2020-10-09 2023-04-27 Synthorx Inc Terapia de combinacion de inmunoncologia con conjugados de il-2 y pembrolizumab.
WO2022079719A1 (en) 2020-10-15 2022-04-21 Yeda Research And Development Co. Ltd. Method of treating myeloid malignancies
EP4232455A2 (en) 2020-10-20 2023-08-30 Sanofi Novel ligands for asialoglycoprotein receptor
CA3198823A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
EP4232582A1 (en) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
WO2022087475A1 (en) 2020-10-23 2022-04-28 The Scripps Research Institute Reverse transcription of polynucleotides comprising unnatural nucleotides
TW202237841A (zh) 2020-11-13 2022-10-01 美商艾拉倫製藥股份有限公司 凝血因子Ｖ（Ｆ５）ｉＲＮＡ組成物及其使用方法
WO2022109139A1 (en) 2020-11-18 2022-05-27 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating angiotensinogen expression
US11987795B2 (en) 2020-11-24 2024-05-21 The Broad Institute, Inc. Methods of modulating SLC7A11 pre-mRNA transcripts for diseases and conditions associated with expression of SLC7A11
EP4256053A1 (en) 2020-12-01 2023-10-11 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
EP4259795A1 (en) 2020-12-08 2023-10-18 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
IL303800A (en) 2020-12-18 2023-08-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating factor xii
WO2022140702A1 (en) 2020-12-23 2022-06-30 Flagship Pioneering, Inc. Compositions of modified trems and uses thereof
WO2022150260A1 (en) 2021-01-05 2022-07-14 Alnylam Pharmaceuticals, Inc. COMPLEMENT COMPONENT 9 (C9) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2022174102A1 (en) 2021-02-12 2022-08-18 Synthorx, Inc. Lung cancer combination therapy with il-2 conjugates and an anti-pd-1 antibody or antigen-binding fragment thereof
EP4291654A2 (en) 2021-02-12 2023-12-20 Alnylam Pharmaceuticals, Inc. Superoxide dismutase 1 (sod1) irna compositions and methods of use thereof for treating or preventing superoxide dismutase 1- (sod1-) associated neurodegenerative diseases
WO2022174101A1 (en) 2021-02-12 2022-08-18 Synthorx, Inc. Skin cancer combination therapy with il-2 conjugates and cemiplimab
WO2022182864A1 (en) 2021-02-25 2022-09-01 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods and methods of use thereof
WO2022182574A1 (en) 2021-02-26 2022-09-01 Alnylam Pharmaceuticals, Inc. KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
MX2023010249A (es) 2021-03-04 2023-09-15 Alnylam Pharmaceuticals Inc Composiciones de arni de proteina similar a la angiopoyetina 3 (angptl3) y metodos para usarlas.
WO2022192519A1 (en) 2021-03-12 2022-09-15 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
US20220288181A1 (en) 2021-03-12 2022-09-15 Northwestern University Antiviral vaccines using spherical nucleic acids
AU2022246786A1 (en) 2021-03-29 2023-10-05 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
WO2022212153A1 (en) 2021-04-01 2022-10-06 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
JP2024517686A (ja) 2021-04-26 2024-04-23 アルナイラム ファーマシューティカルズ， インコーポレイテッド 膜貫通プロテアーゼ、セリン６（ＴＭＰＲＳＳ６）ｉＲＮＡ組成物およびその使用方法
EP4330396A1 (en) 2021-04-29 2024-03-06 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
EP4334448A1 (en) 2021-05-03 2024-03-13 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (ttr) mediated amyloidosis
JP2024522068A (ja) 2021-05-18 2024-06-11 アルナイラム ファーマシューティカルズ， インコーポレイテッド ナトリウム－グルコース共輸送体２（ｓｇｌｔ２）ｉｒｎａ組成物およびその使用方法
US20240263177A1 (en) 2021-05-20 2024-08-08 Korro Bio, Inc. Methods and Compositions for Adar-Mediated Editing
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
EP4347823A1 (en) 2021-06-02 2024-04-10 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
EP4346904A1 (en) 2021-06-03 2024-04-10 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and cetuximab
CN117561334A (zh) 2021-06-04 2024-02-13 阿尔尼拉姆医药品有限公司 人染色体9开放阅读框72（C9ORF72）iRNA药剂组合物和其使用方法
EP4351541A2 (en) 2021-06-08 2024-04-17 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating or preventing stargardt's disease and/or retinal binding protein 4 (rbp4)-associated disorders
CA3222950A1 (en) 2021-06-11 2022-12-15 Bayer Aktiengesellschaft Type v rna programmable endonuclease systems
EP4101928A1 (en) 2021-06-11 2022-12-14 Bayer AG Type v rna programmable endonuclease systems
AU2022293556A1 (en) 2021-06-18 2024-01-18 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ifnar1 expression
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
WO2023278410A1 (en) 2021-06-29 2023-01-05 Korro Bio, Inc. Methods and compositions for adar-mediated editing
JP2024527304A (ja) 2021-06-30 2024-07-24 アルナイラム ファーマシューティカルズ， インコーポレイテッド アンジオテンシノーゲン（ａｇｔ）関連障害を治療するための方法および組成物
WO2023285431A1 (en) 2021-07-12 2023-01-19 Alia Therapeutics Srl Compositions and methods for allele specific treatment of retinitis pigmentosa
WO2023003805A1 (en) 2021-07-19 2023-01-26 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating subjects having or at risk of developing a non-primary hyperoxaluria disease or disorder
AU2022316139A1 (en) 2021-07-23 2024-01-18 Alnylam Pharmaceuticals, Inc. Beta-catenin (ctnnb1) irna compositions and methods of use thereof
WO2023009687A1 (en) 2021-07-29 2023-02-02 Alnylam Pharmaceuticals, Inc. 3-hydroxy-3-methylglutaryl-coa reductase (hmgcr) irna compositions and methods of use thereof
AR126675A1 (es) 2021-08-03 2023-11-01 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi CONTRA LA TRANSTIRRETINA (TTR) Y SUS MÉTODOS DE USO
WO2023014765A1 (en) 2021-08-04 2023-02-09 Alnylam Pharmaceuticals, Inc. iRNA COMPOSITIONS AND METHODS FOR SILENCING ANGIOTENSINOGEN (AGT)
TW202334413A (zh) 2021-08-13 2023-09-01 美商艾拉倫製藥股份有限公司 第十二因子（Ｆ１２）ｉＲＮＡ組成物及其使用方法
AU2022339821A1 (en) 2021-08-31 2024-03-07 Alnylam Pharmaceuticals, Inc. Cell death-inducing dffa-like effector b (cideb) irna compositions and methods of use thereof
EP4144841A1 (en) 2021-09-07 2023-03-08 Bayer AG Novel small rna programmable endonuclease systems with impoved pam specificity and uses thereof
WO2023044370A2 (en) 2021-09-17 2023-03-23 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
CA3232420A1 (en) 2021-09-20 2023-03-23 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
US20250352667A1 (en) 2021-10-22 2025-11-20 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
TW202334418A (zh) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 杭丁頓（ＨＴＴ）ｉＲＮＡ劑組成物及其使用方法
IL312399A (en) 2021-10-29 2024-06-01 Alnylam Pharmaceuticals Inc Complement factor B (CFB) iRNA compositions and methods of using them
EP4452327A1 (en) 2021-12-20 2024-10-30 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
CA3243006A1 (en) 2021-12-21 2025-02-27 Alia Therapeutics Srl TYPE II CAS PROTEINS AND THEIR APPLICATIONS
WO2023122750A1 (en) 2021-12-23 2023-06-29 Synthorx, Inc. Cancer combination therapy with il-2 conjugates and cetuximab
WO2023118068A1 (en) 2021-12-23 2023-06-29 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
EP4469575A2 (en) 2022-01-24 2024-12-04 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
CA3245130A1 (en) 2022-03-01 2023-09-07 Crispr Therapeutics Ag METHODS AND COMPOSITIONS FOR THE TREATMENT OF DISORDERS RELATED TO ANGIOPOIETIN-TYPE PROTEIN 3 (ANGPTL3)
CN118891266A (zh) 2022-03-18 2024-11-01 迪克纳制药公司 使用Mn（II）或Mn（III）试剂用于合成4’-乙酰氧基-核苷的脱羧乙酰氧基化及其用于合成相应的4’-(二甲氧基磷酰基)甲氧基-核苷酸的用途
WO2023194359A1 (en) 2022-04-04 2023-10-12 Alia Therapeutics Srl Compositions and methods for treatment of usher syndrome type 2a
CN120077130A (zh) 2022-08-18 2025-05-30 阿尔尼拉姆医药品有限公司 通用非靶向sirna组合物及其使用方法
WO2024059165A1 (en) 2022-09-15 2024-03-21 Alnylam Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
WO2024056880A2 (en) 2022-09-16 2024-03-21 Alia Therapeutics Srl Enqp type ii cas proteins and applications thereof
IL319629A (en) 2022-09-23 2025-05-01 Ionis Pharmaceuticals Inc Compounds and methods for reducing MECP2 expression
WO2024105162A1 (en) 2022-11-16 2024-05-23 Alia Therapeutics Srl Type ii cas proteins and applications thereof
EP4634376A2 (en) 2022-12-13 2025-10-22 Bayer Aktiengesellschaft Engineered type v rna programmable endonucleases and their uses
WO2024136899A1 (en) 2022-12-21 2024-06-27 Synthorx, Inc. Cancer therapy with il-2 conjugates and chimeric antigen receptor therapies
CN120882867A (zh) 2023-01-11 2025-10-31 阿利亚治疗有限公司 Ii型cas蛋白及其应用
AR131799A1 (es) 2023-02-09 2025-04-30 Alnylam Pharmaceuticals Inc Moléculas de reversir y métodos para su uso
EP4665865A1 (en) 2023-02-17 2025-12-24 Anjarium Biosciences AG Methods of making dna molecules and compositions and uses thereof
TW202444349A (zh) 2023-03-20 2024-11-16 美商欣爍克斯公司 使用ｉｌ－２綴合物之癌症療法
WO2024220746A2 (en) 2023-04-21 2024-10-24 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting fatty acid synthase and related methods
WO2024226499A1 (en) 2023-04-24 2024-10-31 The Broad Institute, Inc. Compositions and methods for modifying fertility
WO2025003344A1 (en) 2023-06-28 2025-01-02 Alia Therapeutics Srl Type ii cas proteins and applications thereof
WO2025015335A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025015338A1 (en) 2023-07-13 2025-01-16 Korro Bio, Inc. Rna-editing oligonucleotides and uses thereof
WO2025024334A1 (en) 2023-07-21 2025-01-30 Marrow Therapeutics, Inc. Hematopoietic cell targeting conjugates and related methods
TW202519653A (zh) 2023-07-25 2025-05-16 美商旗艦先鋒創新有限責任（Ｖｉｉ）公司 Ｃａｓ內切酶及相關方法
US20250092426A1 (en) 2023-07-25 2025-03-20 Flagship Pioneering Innovations Vii, Llc Cas endonucleases and related methods
WO2025034422A1 (en) 2023-08-04 2025-02-13 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating ctnnb1-associated disorders
WO2025059113A1 (en) 2023-09-12 2025-03-20 The Board Of Regents Of The University Of Oklahoma Treatments for enhancing immune response to clostridioides difficile infections
WO2025072331A1 (en) 2023-09-26 2025-04-03 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025072699A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Aminoglycosides for delivery of agents to the kidney
WO2025072672A2 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Slc6a19-targeting modulatory nucleic acid agents
WO2025072713A1 (en) 2023-09-27 2025-04-03 Judo Bio, Inc. Polymyxins for delivery of agents to the kidney
WO2025076031A2 (en) 2023-10-03 2025-04-10 Alnylam Pharmaceuticals, Inc. Peritoneal macrophages comprising a nanoparticle encapsulating a nucleic acid molecule and methods of use thereof
WO2025076291A1 (en) 2023-10-06 2025-04-10 Bluerock Therapeutics Lp Engineered type v rna programmable endonucleases and their uses
WO2025096809A1 (en) 2023-10-31 2025-05-08 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
WO2025117877A2 (en) 2023-12-01 2025-06-05 Flagship Pioneering Innovations Vii, Llc Cas nucleases and related methods
WO2025128799A1 (en) 2023-12-12 2025-06-19 Korro Bio, Inc. Double-stranded rna-editing oligonucleotides and uses thereof
WO2025158385A1 (en) 2024-01-25 2025-07-31 Genzyme Corporation Pegylated il-2 for suppressing adaptive immune response to gene therapy
US20250263702A1 (en) 2024-02-19 2025-08-21 Flagship Pioneering Innovations Vii, Llc Rnai agents targeting cideb and related methods
US20250313840A1 (en) 2024-03-20 2025-10-09 Vertex Pharmaceuticals Incorporated Mucin-5b (muc5b) targeted sirna and antisense oligonucleotides and methods of use thereof
WO2025207517A2 (en) 2024-03-25 2025-10-02 Synthorx, Inc. Synthetic trna synthetases and cells comprising synthetic molecules for production of polypeptides
WO2025210147A1 (en) 2024-04-04 2025-10-09 Alia Therapeutics Srl Type v cas proteins and applications thereof
WO2025217275A2 (en) 2024-04-10 2025-10-16 Flagship Pioneering Innovations Vii, Llc Immune cell targeted compositions and related methods
WO2025259743A1 (en) 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dual conjugate compounds for extrahepatic delivery
WO2025259747A2 (en) 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS5927900A (ja) * 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
US4904582A (en) * 1987-06-11 1990-02-27 Synthetic Genetics Novel amphiphilic nucleic acid conjugates
WO1989005358A1 (en) * 1987-11-30 1989-06-15 University Of Iowa Research Foundation Dna and rna molecules stabilized by modifications of the 3'-terminal phosphodiester linkage and their use as nucleic acid probes and as therapeutic agents to block the expression of specifically targeted genes
CA2028153A1 (en) * 1989-02-24 1990-08-25 Steven S. Smith Heterologous block oligomers
WO1991006629A1 (en) * 1989-10-24 1991-05-16 Gilead Sciences, Inc. Oligonucleotide analogs with novel linkages
US5223618A (en) * 1990-08-13 1993-06-29 Isis Pharmaceuticals, Inc. 4'-desmethyl nucleoside analog compounds
US5378825A (en) * 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5489677A (en) * 1990-07-27 1996-02-06 Isis Pharmaceuticals, Inc. Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms
US5138045A (en) * 1990-07-27 1992-08-11 Isis Pharmaceuticals Polyamine conjugated oligonucleotides
PT98562B (pt) * 1990-08-03 1999-01-29 Sanofi Sa Processo para a preparacao de composicoes que compreendem sequencias de nucleo-sidos com cerca de 6 a cerca de 200 bases resistentes a nucleases
US5245022A (en) * 1990-08-03 1993-09-14 Sterling Drug, Inc. Exonuclease resistant terminally substituted oligonucleotides
US5214134A (en) * 1990-09-12 1993-05-25 Sterling Winthrop Inc. Process of linking nucleosides with a siloxane bridge
US5389023A (en) * 1993-03-22 1995-02-14 Mcintyre; Jonothon M. W. Body surfing board

Also Published As

Publication number Publication date
DK0541722T3 (da) 1996-04-22
KR930702372A (ko) 1993-09-08
FI930455A7 (fi) 1993-03-24
HU211668A9 (en) 1995-12-28
ES2083593T3 (es) 1996-04-16
US5677439A (en) 1997-10-14
IL99066A (en) 1996-01-31
WO1992002534A3 (en) 1992-06-11
FI930455A0 (fi) 1993-02-02
FI930455L (fi) 1993-03-24
HUT67834A (en) 1995-05-29
EP0541722B1 (en) 1995-12-20
IL121421A0 (en) 1998-01-04
PT98562A (pt) 1992-06-30
PT98562B (pt) 1999-01-29
ATE131827T1 (de) 1996-01-15
MY107332A (en) 1995-11-30
DE69115702T2 (de) 1996-06-13
TW222641B (enExample) 1994-04-21
JPH06502300A (ja) 1994-03-17
CA2088673A1 (en) 1992-02-04
HU217036B (hu) 1999-11-29
EP0541722A1 (en) 1993-05-19
AU8521791A (en) 1992-03-02
AU667459B2 (en) 1996-03-28
BR9106729A (pt) 1993-07-20
IL99066A0 (en) 1992-07-15
AU2008195A (en) 1995-10-12
NZ239247A (en) 1993-11-25
AU692532B2 (en) 1998-06-11
WO1992002534A2 (en) 1992-02-20
HU9300282D0 (en) 1993-04-28
IL113519A0 (en) 1995-07-31
IL113519A (en) 1997-11-20
GR3018881T3 (en) 1996-05-31
DE69115702D1 (de) 1996-02-01

Similar Documents

Publication Publication Date Title
KR100211552B1 (ko) 유전자 발현 억제용 화합물 및 방법
IE912761A1 (en) Compounds and methods for inhibiting gene expression
US6031086A (en) Antisense oligonucleitide containing compositions and method of forming duplexes
JP3756313B2 (ja) 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
RU2079508C1 (ru) Способ соединения нуклеозидов 3'-5'-межнуклеотидным силильным звеном
US20040132684A1 (en) Polymeric nucleoside prodrugs
JP7263236B2 (ja) 新規二環式ヌクレオシドおよびそれから調製されたオリゴマー
EP0691979A1 (en) Novel 5'-substituted nucleosides and oligomers produced therefrom
JPH06220083A (ja) オリゴヌクレオチドとオリゴヌクレオチド類似体の固相合成のための方法と化合物
CZ129197A3 (en) Nucleosides with modified sugar portion of a molecule and their use for preparing oligonucleotides
KR19990022711A (ko) 올리고누클레오티드 합성을 위한 개선된 방법
US5892024A (en) Bifunctional nucleosides, oligomers thereof, and methods of making and using the same
JP7719788B2 (ja) 核酸オリゴマーの製造方法
EP0611075B1 (en) Modified oligodeoxyribonucleotides, their preparation and their therapeutic use
EP1332150A1 (en) Modified nucleosides and nucleotides and use thereof
RU2131436C1 (ru) Резистентные к нуклеазе олигонуклеозиды, способ их получения и резистентный к нуклеазе нуклеозидный димер
WO1995031470A2 (en) Antisense inhibitors of gene expression
JP2000505081A (ja) オリゴヌクレオチドｎ３’→ｐ５’ホスホルアミデートの固相合成
FR2612930A1 (fr) Sondes oligonucleotidiques a

Legal Events

Date Code Title Description
PA0105 International application

St.27 status event code: A-0-1-A10-A15-nap-PA0105

R17-X000 Change to representative recorded

St.27 status event code: A-3-3-R10-R17-oth-X000

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

A201 Request for examination
PA0201 Request for examination

St.27 status event code: A-1-2-D10-D11-exm-PA0201

N231 Notification of change of applicant
PN2301 Change of applicant

St.27 status event code: A-3-3-R10-R13-asn-PN2301

St.27 status event code: A-3-3-R10-R11-asn-PN2301

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

St.27 status event code: A-1-2-D10-D21-exm-PE0902

P11-X000 Amendment of application requested

St.27 status event code: A-2-2-P10-P11-nap-X000

P13-X000 Application amended

St.27 status event code: A-2-2-P10-P13-nap-X000

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

E701 Decision to grant or registration of patent right
PE0701 Decision of registration

St.27 status event code: A-1-2-D10-D22-exm-PE0701

PN2301 Change of applicant

St.27 status event code: A-3-3-R10-R13-asn-PN2301

St.27 status event code: A-3-3-R10-R11-asn-PN2301

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

PN2301 Change of applicant

St.27 status event code: A-3-3-R10-R13-asn-PN2301

St.27 status event code: A-3-3-R10-R11-asn-PN2301

R18-X000 Changes to party contact information recorded

St.27 status event code: A-3-3-R10-R18-oth-X000

GRNT Written decision to grant
PN2301 Change of applicant

St.27 status event code: A-5-5-R10-R13-asn-PN2301

St.27 status event code: A-5-5-R10-R11-asn-PN2301

PR0701 Registration of establishment

St.27 status event code: A-2-4-F10-F11-exm-PR0701

PR1002 Payment of registration fee

St.27 status event code: A-2-2-U10-U12-oth-PR1002

Fee payment year number: 1

R17-X000 Change to representative recorded

St.27 status event code: A-5-5-R10-R17-oth-X000

R18-X000 Changes to party contact information recorded

St.27 status event code: A-5-5-R10-R18-oth-X000

PG1601 Publication of registration

St.27 status event code: A-4-4-Q10-Q13-nap-PG1601

R18-X000 Changes to party contact information recorded

St.27 status event code: A-5-5-R10-R18-oth-X000

R18-X000 Changes to party contact information recorded

St.27 status event code: A-5-5-R10-R18-oth-X000

LAPS Lapse due to unpaid annual fee
PC1903 Unpaid annual fee

St.27 status event code: A-4-4-U10-U13-oth-PC1903

Not in force date: 20020505

Payment event data comment text: Termination Category : DEFAULT_OF_REGISTRATION_FEE

PC1903 Unpaid annual fee

St.27 status event code: N-4-6-H10-H13-oth-PC1903

Ip right cessation event data comment text: Termination Category : DEFAULT_OF_REGISTRATION_FEE

Not in force date: 20020505