JPS5927900A - 固定化オリゴヌクレオチド - Google Patents

固定化オリゴヌクレオチド

Info

Publication number
JPS5927900A
JPS5927900A JP57138136A JP13813682A JPS5927900A JP S5927900 A JPS5927900 A JP S5927900A JP 57138136 A JP57138136 A JP 57138136A JP 13813682 A JP13813682 A JP 13813682A JP S5927900 A JPS5927900 A JP S5927900A
Authority
JP
Japan
Prior art keywords
group
oligonucleotide
compound
sepharose
derivative
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.)
Granted
Application number
JP57138136A
Other languages
English (en)
Japanese (ja)
Other versions
JPH0372639B2 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html
Inventor
Kenichi Miyoshi
健一 三好
Toru Fuwa
不破　亨
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Wakunaga Pharmaceutical Co Ltd
Original Assignee
Wakunaga Pharmaceutical Co Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Wakunaga Pharmaceutical Co Ltd filed Critical Wakunaga Pharmaceutical Co Ltd
Priority to JP57138136A priority Critical patent/JPS5927900A/ja
Priority to DE8383107730T priority patent/DE3373817D1/de
Priority to EP83107730A priority patent/EP0101985B1/en
Priority to CA000434126A priority patent/CA1202254A/en
Publication of JPS5927900A publication Critical patent/JPS5927900A/ja
Priority to US06/790,658 priority patent/US4667025A/en
Priority to US07/016,889 priority patent/US4820812A/en
Priority to US07/016,835 priority patent/US4789737A/en
Publication of JPH0372639B2 publication Critical patent/JPH0372639B2/ja
Granted 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
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02P—CLIMATE CHANGE MITIGATION TECHNOLOGIES IN THE PRODUCTION OR PROCESSING OF GOODS
    • Y02P20/00—Technologies relating to chemical industry
    • Y02P20/50—Improvements relating to the production of bulk chemicals
    • Y02P20/55—Design of synthesis routes, e.g. reducing the use of auxiliary or protecting groups

Landscapes

  • Chemical & Material Sciences (AREA)
  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Saccharide Compounds (AREA)
  • Treatment Of Liquids With Adsorbents In General (AREA)
JP57138136A 1982-08-09 1982-08-09 固定化オリゴヌクレオチド Granted JPS5927900A (ja)

Priority Applications (7)

Application Number Priority Date Filing Date Title
JP57138136A JPS5927900A (ja) 1982-08-09 1982-08-09 固定化オリゴヌクレオチド
DE8383107730T DE3373817D1 (en) 1982-08-09 1983-08-05 Oligonucleotide derivatives and production thereof
EP83107730A EP0101985B1 (en) 1982-08-09 1983-08-05 Oligonucleotide derivatives and production thereof
CA000434126A CA1202254A (en) 1982-08-09 1983-08-08 Oligonucleotide derivatives and production thereof
US06/790,658 US4667025A (en) 1982-08-09 1985-10-24 Oligonucleotide derivatives
US07/016,889 US4820812A (en) 1982-08-09 1987-02-20 Oligonucleotide derivatives and production thereof
US07/016,835 US4789737A (en) 1982-08-09 1987-02-20 Oligonucleotide derivatives and production thereof

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
JP57138136A JPS5927900A (ja) 1982-08-09 1982-08-09 固定化オリゴヌクレオチド

Related Child Applications (3)

Application Number Title Priority Date Filing Date
JP58204306A Division JPS5993100A (ja) 1983-10-31 1983-10-31 オリゴヌクレオチド誘導体
JP58204305A Division JPS5993099A (ja) 1983-10-31 1983-10-31 オリゴヌクレオチド誘導体およびその製造法
JP58204304A Division JPS5993098A (ja) 1983-10-31 1983-10-31 オリゴヌクレオチド誘導体およびその製造法

Publications (2)

Publication Number Publication Date
JPS5927900A true JPS5927900A (ja) 1984-02-14
JPH0372639B2 JPH0372639B2 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html) 1991-11-19

Family

ID=15214830

Family Applications (1)

Application Number Title Priority Date Filing Date
JP57138136A Granted JPS5927900A (ja) 1982-08-09 1982-08-09 固定化オリゴヌクレオチド

Country Status (5)

Country Link
US (3) US4667025A (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
EP (1) EP0101985B1 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
JP (1) JPS5927900A (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
CA (1) CA1202254A (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
DE (1) DE3373817D1 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS59130221A (ja) * 1982-10-26 1984-07-26 シ−テイエイ・フイナンツ・アクチエンゲゼルシヤフト ヒトと動物の遺伝学的変動域内における器官の組織物質の改良剤と改良方法
US7491857B2 (en) 2005-07-27 2009-02-17 National Institute Of Advanced Industrial Science And Technology Oligonucleotide probe

Families Citing this family (612)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA1223831A (en) 1982-06-23 1987-07-07 Dean Engelhardt Modified nucleotides, methods of preparing and utilizing and compositions containing the same
JPS5927900A (ja) * 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
US5821058A (en) * 1984-01-16 1998-10-13 California Institute Of Technology Automated DNA sequencing technique
USRE43096E1 (en) 1984-01-16 2012-01-10 California Institute Of Technology Tagged extendable primers and extension products
JPS61112093A (ja) * 1984-11-05 1986-05-30 Wakunaga Seiyaku Kk ヌクレオチド誘導体
DE3538433A1 (de) * 1985-10-29 1987-05-14 Consortium Elektrochem Ind Dna-fragment mit dem cyclodextrin-glycosyl-transferase-strukturgen, expressionsvektor, mikroorganismen zur expression und herstellungsverfahren
EP0289619B1 (en) * 1986-10-30 1995-08-16 Daicel Chemical Industries, Ltd. Process for synthesizing oligonucleotides and compounds for forming high-molecular protective group
US6270961B1 (en) * 1987-04-01 2001-08-07 Hyseq, Inc. Methods and apparatus for DNA sequencing and DNA identification
US4923901A (en) * 1987-09-04 1990-05-08 Millipore Corporation Membranes with bound oligonucleotides and peptides
JPH084519B2 (ja) * 1987-12-25 1996-01-24 エフ・ホフマン―ラ ロシュ アーゲー ハイブリッド形成用担体およびその調整方法
JPH0291088A (ja) * 1988-09-29 1990-03-30 Central Glass Co Ltd ホスファイトおよびヌクレオシド‐３’‐ホスファイト誘導体およびこれを用いるオリゴヌクレオチドの合成法
US5176996A (en) * 1988-12-20 1993-01-05 Baylor College Of Medicine Method for making synthetic oligonucleotides which bind specifically to target sites on duplex DNA molecules, by forming a colinear triplex, the synthetic oligonucleotides and methods of use
JPH03184992A (ja) * 1989-09-14 1991-08-12 Tosoh Corp オリゴヌクレオチド誘導体及びその製造方法
IL113519A (en) * 1990-08-03 1997-11-20 Sterling Winthrop Inc Oligonucleoside sequences of from about 6 to about 200 bases having a three atom internucleoside linkage, their preparation and pharmaceutical compositions for inhibiting gene expression containing said oligonucleosides
AU1248292A (en) * 1990-12-06 1992-07-08 Affymax Technologies N.V. Sequencing by hybridization of a target nucleic acid to a matrix of defined oligonucleotides
DK51092D0 (da) * 1991-05-24 1992-04-15 Ole Buchardt Oligonucleotid-analoge betegnet pna, monomere synthoner og fremgangsmaade til fremstilling deraf samt anvendelser deraf
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
TW323284B (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html) * 1992-03-23 1997-12-21 Novartis Ag
IL107150A0 (en) * 1992-09-29 1993-12-28 Isis Pharmaceuticals Inc Oligonucleotides having a conserved g4 core sequence
US5583211A (en) * 1992-10-29 1996-12-10 Beckman Instruments, Inc. Surface activated organic polymers useful for location - specific attachment of nucleic acids, peptides, proteins and oligosaccharides
CA2143428A1 (en) * 1993-07-09 1995-01-19 Takanori Oka Method of nucleic acid-differentiation and assay kit for nucleic acid-differentiation
EP0728139B1 (en) 1993-09-03 2003-08-13 Isis Pharmaceuticals, Inc. Amine-derivatized nucleosides and oligonucleosides
KR100361933B1 (ko) * 1993-09-08 2003-02-14 라 졸라 파마슈티칼 컴파니 화학적으로정의된비중합성결합가플랫폼분자및그것의콘주게이트
US6214987B1 (en) 1994-09-02 2001-04-10 Andrew C. Hiatt Compositions for enzyme catalyzed template-independent formation of phosphodiester bonds using protected nucleotides
US5990300A (en) * 1994-09-02 1999-11-23 Andrew C. Hiatt Enzyme catalyzed template-independent creation of phosphodiester bonds using protected nucleotides
US6232465B1 (en) 1994-09-02 2001-05-15 Andrew C. Hiatt Compositions for enzyme catalyzed template-independent creation of phosphodiester bonds using protected nucleotides
US5808045A (en) * 1994-09-02 1998-09-15 Andrew C. Hiatt Compositions for enzyme catalyzed template-independent creation of phosphodiester bonds using protected nucleotides
US5872244A (en) * 1994-09-02 1999-02-16 Andrew C. Hiatt 3' protected nucleotides for enzyme catalyzed template-independent creation of phosphodiester bonds
US5763594A (en) * 1994-09-02 1998-06-09 Andrew C. Hiatt 3' protected nucleotides for enzyme catalyzed template-independent creation of phosphodiester bonds
US6420549B1 (en) 1995-06-06 2002-07-16 Isis Pharmaceuticals, Inc. Oligonucleotide analogs having modified dimers
WO2005121370A2 (en) * 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Oligomeric compounds that facilitate risc loading
US20070275921A1 (en) * 1996-06-06 2007-11-29 Isis Pharmaceuticals, Inc. Oligomeric Compounds That Facilitate Risc Loading
US20030044941A1 (en) 1996-06-06 2003-03-06 Crooke Stanley T. Human RNase III and compositions and uses thereof
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
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US20040266706A1 (en) * 2002-11-05 2004-12-30 Muthiah Manoharan Cross-linked oligomeric compounds and their use in gene modulation
US20050042647A1 (en) * 1996-06-06 2005-02-24 Baker Brenda F. Phosphorous-linked oligomeric compounds and their use in gene modulation
CA2294988C (en) 1997-07-01 2015-11-24 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
WO1999060167A1 (en) * 1998-05-21 1999-11-25 Isis Pharmaceuticals, Inc. Compositions and methods for topical delivery of oligonucleotides
WO1999060012A1 (en) * 1998-05-21 1999-11-25 Isis Pharmaceuticals, Inc. Compositions and methods for non-parenteral delivery of oligonucleotides
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
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
US6656730B1 (en) 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
US6921638B2 (en) * 1999-06-25 2005-07-26 Amersham Biosciences Ab Hydrogel-based microarray signal amplification methods and devices therefor
JP2003508763A (ja) * 1999-08-27 2003-03-04 マトリックス テクノロジーズ コーポレイション 固体支持体上にリガンドを固定化する方法及び装置並びにその使用方法
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
US20030176385A1 (en) * 2000-02-15 2003-09-18 Jingfang Ju Antisense modulation of protein expression
US6680172B1 (en) 2000-05-16 2004-01-20 Regents Of The University Of Michigan Treatments and markers for cancers of the central nervous system
JP2003535208A (ja) * 2000-06-08 2003-11-25 ラ ホヤ ファーマシューティカル カンパニー 高分子量ポリエチレン・オキシドを含む多価プラットホーム分子
US8568766B2 (en) 2000-08-24 2013-10-29 Gattadahalli M. Anantharamaiah Peptides and peptide mimetics to treat pathologies associated with eye disease
EP1326892A2 (en) 2000-10-12 2003-07-16 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
US20030170675A1 (en) * 2001-04-11 2003-09-11 The Gov't Of The U.S Of America As Represented By The Secretary Of The Dept. Of Health & Human Serv. Methods of manipulating nucleic acids
US20050221304A1 (en) * 2001-04-11 2005-10-06 Charlie Xiang Modified random primers for probe labeling
EP1392272B1 (en) 2001-05-11 2014-08-06 Merrion Research III Limited Permeation enhancers
DK2000545T3 (da) 2001-06-20 2011-11-28 Genentech Inc Sammensætninger og fremgangsmåder til diagnose og behandling af lunge-tumor
US7803915B2 (en) * 2001-06-20 2010-09-28 Genentech, Inc. Antibody compositions for the diagnosis and treatment of tumor
US20050107595A1 (en) * 2001-06-20 2005-05-19 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
WO2003000707A2 (en) 2001-06-21 2003-01-03 Isis Pharmaceuticals, Inc. Antisense modulation of superoxide dismutase 1, soluble expression
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
US7227014B2 (en) 2001-08-07 2007-06-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein (a) expression
DE60238143D1 (de) 2001-09-18 2010-12-09 Genentech Inc Zusammensetzungen und verfahren für die diagnose von tumoren
US6750019B2 (en) 2001-10-09 2004-06-15 Isis Pharmaceuticals, Inc. Antisense modulation of insulin-like growth factor binding protein 5 expression
EP2270231A3 (en) 2001-10-09 2011-04-27 ISIS Pharmaceuticals, Inc. Antisense modulation of insulin-like growth factor binding protein 5 expression
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
NZ556415A (en) 2002-01-02 2009-02-28 Genentech Inc Compositions and methods for the diagnosis and treatment of glioma tumor
US20030180712A1 (en) 2002-03-20 2003-09-25 Biostratum Ab Inhibition of the beta3 subunit of L-type Ca2+ channels
EP2011886A3 (en) 2002-04-16 2009-02-11 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
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
US20040092470A1 (en) * 2002-06-18 2004-05-13 Leonard Sherry A. Dry powder oligonucleotide formualtion, preparation and its uses
US20040019000A1 (en) * 2002-07-19 2004-01-29 Muthiah Manoharan Polyalkyleneamine-containing oligomers
WO2004013160A2 (en) 2002-08-05 2004-02-12 University Of Rochester Protein transducing domain/deaminase chimeric proteins, related compounds, and uses thereof
US6878805B2 (en) * 2002-08-16 2005-04-12 Isis Pharmaceuticals, Inc. Peptide-conjugated oligomeric compounds
CA2498777C (en) 2002-09-13 2015-01-13 Replicor, Inc. Non-sequence complementary antiviral oligonucleotides
EP2272958A1 (en) 2002-09-26 2011-01-12 ISIS Pharmaceuticals, Inc. Modulation of forkhead box O1A expression
WO2004110345A2 (en) * 2002-10-29 2004-12-23 Pharmacia Corporation Differentially expressed genes involved in cancer, the polypeptides encoded thereby, and methods of using the same
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
US9150605B2 (en) 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
CA2504720C (en) 2002-11-05 2013-12-24 Isis Pharmaceuticals, Inc. Chimeric oligomeric compounds and their use in gene modulation
EP1560840B1 (en) 2002-11-05 2015-05-06 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
AU2003294281B2 (en) 2002-11-13 2010-05-20 Kastle Therapeutics, Llc Antisense modulation of apolipoprotein B expression
PT2336318E (pt) 2002-11-13 2013-06-12 Genzyme Corp Modulação anti-sentido da expressão de apolipoproteína b
WO2004046330A2 (en) 2002-11-15 2004-06-03 Morphotek, Inc. Methods of generating high-production of antibodies from hybridomas created by in vitro immunization
EP2292259A3 (en) 2002-11-15 2011-03-23 MUSC Foundation For Research Development Complement receptor 2 targeted complement modulators
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
DK1597366T3 (da) 2003-02-11 2013-02-25 Antisense Therapeutics Ltd Modulering af ekspression af insulin-lignende vækstfaktor receptor I
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
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
JP4579911B2 (ja) 2003-06-03 2010-11-10 アイシス・ファーマシューティカルズ・インコーポレイテッド スルビビン発現の調節
US7786290B2 (en) 2003-06-13 2010-08-31 Alnylam Pharmaceuticals, Inc. Double-stranded ribonucleic acid with increased effectiveness in an organism
WO2004113496A2 (en) * 2003-06-20 2004-12-29 Isis Pharmaceuticals, Inc. Double stranded compositions comprising a 3’-endo modified strand for use in gene modulation
WO2005013901A2 (en) 2003-07-31 2005-02-17 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for use in modulation of small non-coding rnas
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
US20070123480A1 (en) * 2003-09-11 2007-05-31 Replicor Inc. Oligonucleotides targeting prion diseases
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
EP2256200A3 (en) 2003-09-18 2012-07-18 Isis Pharmaceuticals, Inc. Modulation of eIF4E expression
EP1678194B1 (en) 2003-10-10 2013-06-26 Alchemia Oncology Pty Limited The modulation of hyaluronan synthesis and degradation in the treatment of disease
US20050191653A1 (en) 2003-11-03 2005-09-01 Freier Susan M. Modulation of SGLT2 expression
NZ576411A (en) 2003-11-17 2010-04-30 Genentech Inc Compositions and methods for the treatment of tumor of hematopoietic origin
EP1711606A2 (en) 2004-01-20 2006-10-18 Isis Pharmaceuticals, Inc. Modulation of glucocorticoid receptor expression
US8778900B2 (en) * 2004-01-22 2014-07-15 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP1 expression
US7468431B2 (en) * 2004-01-22 2008-12-23 Isis Pharmaceuticals, Inc. Modulation of eIF4E-BP2 expression
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
EP1730309B1 (en) 2004-03-15 2016-05-04 Ionis Pharmaceuticals, Inc. Compositions and methods for optimizing cleavage of rna by rnase h
EP1737878A2 (en) 2004-04-05 2007-01-03 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
US20050260755A1 (en) * 2004-04-06 2005-11-24 Isis Pharmaceuticals, Inc. Sequential delivery of oligomeric compounds
CA2562151C (en) 2004-04-30 2016-09-06 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising a c5-modified pyrimidine
AU2005326817B2 (en) 2004-05-21 2012-04-19 Benaroya Research Institute 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
US20090048192A1 (en) * 2004-06-03 2009-02-19 Isis Pharmaceuticals, Inc. Double Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
JP2008501335A (ja) * 2004-06-03 2008-01-24 アイシス ファーマシューティカルズ、インク． キメラギャップ化オリゴマー組成物
WO2006023880A2 (en) * 2004-08-23 2006-03-02 Isis Pharmaceuticals, Inc. Compounds and methods for the characterization of oligonucleotides
US7884086B2 (en) 2004-09-08 2011-02-08 Isis Pharmaceuticals, Inc. Conjugates for use in hepatocyte free uptake assays
ES2729826T3 (es) * 2004-09-23 2019-11-06 Arc Medical Devices Inc Composiciones farmacéuticas y métodos relacionados para inhibir adherencias fibrosas o enfermedad inflamatoria usando fucanos con bajo contenido de sulfato
WO2006098998A2 (en) 2005-03-10 2006-09-21 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
EP1891141B1 (en) 2005-05-31 2016-11-16 Ecole Polytechnique Fédérale de Lausanne (EPFL) Triblock copolymers for cytoplasmic delivery of gene-based drugs
WO2006138145A1 (en) 2005-06-14 2006-12-28 Northwestern University Nucleic acid functionalized nanoparticles for therapeutic applications
RU2292350C1 (ru) * 2005-10-03 2007-01-27 Институт цитологии и генетики Сибирского отделения Российской академии наук (СО РАН) Способ получения 5`-аминоалкилфосфат олигодезоксирибонуклеотидов
US8080534B2 (en) 2005-10-14 2011-12-20 Phigenix, Inc Targeting PAX2 for the treatment of breast cancer
EP2395076A1 (en) 2005-10-14 2011-12-14 MUSC Foundation For Research Development Targeting PAX2 for the induction of DEFB1-mediated tumor immunity and cancer therapy
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
US20100069461A1 (en) 2005-11-09 2010-03-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor v leiden mutant gene
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
KR101547579B1 (ko) 2006-03-31 2015-08-27 알닐람 파마슈티칼스 인코포레이티드 Ｅｇ5 유전자의 발현을 억제하는 이본쇄 리보핵산
BRPI0711182A2 (pt) * 2006-05-03 2011-08-23 Baltic Technology Dev agentes anti-sentido combinando base fortemente ligada-oligonucleotìdeo modificado e nuclease artificial
CN101489566B (zh) 2006-05-19 2012-04-18 阿尔尼拉姆医药品有限公司 Aha基因的RNAi调控及其治疗性应用
CA2653451C (en) 2006-05-22 2015-12-29 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of ikk-b gene
EP2023938A4 (en) * 2006-05-23 2010-11-10 Isis Pharmaceuticals Inc MODULATION OF CHREBP EXPRESSION
US20090280188A1 (en) * 2006-06-23 2009-11-12 Northwestern University Asymmetric functionalizated nanoparticles and methods of use
WO2008011473A2 (en) 2006-07-19 2008-01-24 Isis Pharmaceuticals, Inc. Compositions and their uses directed to hbxip
WO2008136852A2 (en) 2006-11-01 2008-11-13 University Of Rochester Methods and compositions related to the structure and function of apobec3g
EP2104509A4 (en) * 2006-12-11 2010-03-24 Univ Utah Res Found COMPOSITIONS AND METHODS FOR THE TREATMENT OF PATHOLOGICAL ANGIOGENESIS AND VASPERMEABILITY
AU2008212820B2 (en) * 2007-02-09 2014-01-30 Northwestern University Particles for detecting intracellular targets
WO2008121604A2 (en) 2007-03-29 2008-10-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a gene from the ebola
MX2009013046A (es) 2007-05-30 2010-02-17 Univ Northwestern Nanoparticulas con grupo funcional de acido nucleico para aplicaciones terapeuticas.
EP2195340A4 (en) 2007-08-28 2011-05-11 Uab Research Foundation SYNTHETIC APOLIPOPROTEIN E-IMITATING POLYPEPTIDES AND METHOD OF USE
WO2009032693A2 (en) 2007-08-28 2009-03-12 Uab Research Foundation Synthetic apolipoprotein e mimicking polypeptides and methods of use
WO2009039466A1 (en) 2007-09-20 2009-03-26 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
AU2008307482B2 (en) 2007-10-02 2012-07-12 Amgen Inc. Increasing erythropoietin using nucleic acids hybridizable to micro-RNA and precursors thereof
US20100280098A1 (en) * 2007-10-05 2010-11-04 Juliano Rudolph L Receptor targeted oligonucleotides
EP2217705A2 (en) * 2007-11-05 2010-08-18 Baltic Technology Development, Ltd. Use of oligonucleotides with modified bases in hybridization of nucleic acids
WO2009067243A2 (en) 2007-11-20 2009-05-28 Isis Pharmaceuticals Inc. Modulation of cd40 expression
CA2707042A1 (en) 2007-12-10 2009-06-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of factor vii gene
KR101397407B1 (ko) 2008-03-05 2014-06-19 알닐람 파마슈티칼스 인코포레이티드 Ｅｇ5 및 ＶＥＧＦ 유전자의 발현을 억제하기 위한 조성물 및 방법
EP2105145A1 (en) * 2008-03-27 2009-09-30 ETH Zürich Method for muscle-specific delivery lipid-conjugated oligonucleotides
EP2274423A2 (en) * 2008-04-04 2011-01-19 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
JP5788312B2 (ja) 2008-04-11 2015-09-30 アルニラム ファーマスーティカルズ インコーポレイテッドＡｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 標的リガンドをエンドソーム分解性成分と組み合わせることによる核酸の部位特異的送達
US8324366B2 (en) 2008-04-29 2012-12-04 Alnylam Pharmaceuticals, Inc. Compositions and methods for delivering RNAI using lipoproteins
US20110237646A1 (en) * 2008-08-07 2011-09-29 Isis Pharmaceuticals, Inc. Modulation of transthyretin expression for the treatment of cns related disorders
AU2009275387B2 (en) 2008-08-25 2010-07-08 Excaliard Pharmaceuticals, Inc. Antisense oligonucleotides directed against connective tissue growth factor and uses thereof
EP2690175B1 (en) 2008-09-02 2016-12-28 Alnylam Pharmaceuticals, Inc. Compositions and methods for combined inhibition of mutant EGFR gene and IL-6 expression
US10022454B2 (en) 2008-09-23 2018-07-17 Liposciences, Llc Functionalized phosphorodiamites for therapeutic oligonucleotide synthesis
EP3587434A1 (en) 2008-09-23 2020-01-01 Alnylam Pharmaceuticals Inc. Chemical modifications of monomers and oligonucleotides with click components for conjugation with ligands
EP2346883B1 (en) 2008-09-23 2016-03-23 Scott G. Petersen Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating rna interference
US8546554B2 (en) 2008-09-25 2013-10-01 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Serum Amyloid A gene
AU2009307677C1 (en) 2008-10-20 2022-09-22 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of transthyretin
WO2010048585A2 (en) 2008-10-24 2010-04-29 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
DK2355851T3 (en) 2008-11-10 2018-06-25 Arbutus Biopharma Corp Newly known lipids and compositions for release of therapeutic agents
EP3335705A1 (en) 2008-11-24 2018-06-20 Northwestern University Polyvalent rna-nanoparticle compositions
ES2760912T3 (es) 2008-12-04 2020-05-18 Curna Inc Tratamiento de enfermedades relacionadas con la Sirtuina 1 (SIRT1) por inhibición del transcrito antisentido natural de la Sirtuina 1
US8921329B2 (en) 2008-12-04 2014-12-30 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
EP3225281A1 (en) 2008-12-10 2017-10-04 Alnylam Pharmaceuticals, Inc. Gnaq targeted dsrna compositions and methods for inhibiting expression
MX2011007350A (es) * 2009-01-08 2011-09-06 Univ Northwestern Inhibicion de produccion de proteina bacteriana por conjugados de nanoparticulas modificadas por oligonucleotidos polivalentes.
US20100233270A1 (en) 2009-01-08 2010-09-16 Northwestern University Delivery of Oligonucleotide-Functionalized Nanoparticles
WO2010091308A2 (en) 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
JP5766126B2 (ja) 2009-02-12 2015-08-19 クルナ・インコーポレーテッド 脳由来神経栄養因子（ｂｄｎｆ）関連疾病の、ｂｄｎｆに対する天然アンチセンス転写物の抑制による治療
US20110319476A1 (en) 2009-02-12 2011-12-29 Opko Curna, Llc Treatment of glial cell derived neurotrophic factor (gdnf) related diseases by inhibition of natural antisense transcript to gdnf
WO2010099341A1 (en) 2009-02-26 2010-09-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of mig-12 gene
EP3424939A1 (en) 2009-03-02 2019-01-09 Alnylam Pharmaceuticals Inc. Nucleic acid chemical modifications
EP2403946A4 (en) 2009-03-04 2012-11-14 TREATMENT OF SIRTUIN 1 (SIRT1) -HANDLED ILLNESSES BY INHIBITING THE NATURAL ANTISENSE TRANSCRIPT AGAINST SIRT 1
AU2010223967B2 (en) 2009-03-12 2015-07-30 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of Eg5 and VEGF genes
EP2408919B1 (en) 2009-03-16 2017-10-18 CuRNA, Inc. Treatment of nuclear factor (erythroid-derived 2)-like 2 (nrf2) related diseases by inhibition of natural antisense transcript to nrf2
MX2011009752A (es) 2009-03-17 2011-09-29 Opko Curna Llc Tratamiento de enfermedades relacionadas a homologo tipo delta 1(dlk1) por inhibicion de transcrito antisentido natural a homologo tipo delta (dlk1).
EP3248618A1 (en) 2009-04-22 2017-11-29 Massachusetts Institute Of Technology Innate immune suppression enables repeated delivery of long rna molecules
EP2424987B1 (en) 2009-05-01 2017-11-15 CuRNA, Inc. Treatment of hemoglobin (hbf/hbg) related diseases by inhibition of natural antisense transcript to hbf/hbg
CN106237345A (zh) 2009-05-06 2016-12-21 库尔纳公司 通过针对脂质转运和代谢基因的天然反义转录物的抑制治疗脂质转运和代谢基因相关疾病
ES2609655T3 (es) 2009-05-06 2017-04-21 Curna, Inc. Tratamiento de enfermedades relacionadas con tristetraprolina (TTP) mediante inhibición de transcrito antisentido natural para TTP
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
JP5960049B2 (ja) 2009-05-28 2016-08-02 クルナ・インコーポレーテッド 抗ウイルス遺伝子に対する天然アンチセンス転写物の抑制による抗ウイルス遺伝子関連疾患の治療
PL3431076T3 (pl) 2009-06-10 2022-01-31 Arbutus Biopharma Corporation Ulepszona formulacja lipidowa
WO2010148065A2 (en) 2009-06-16 2010-12-23 Curna, Inc. Treatment of paraoxonase 1 (pon1) related diseases by inhibition of natural antisense transcript to pon1
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
SMT202000020T1 (it) 2009-06-17 2020-03-13 Biogen Ma Inc Composizioni e metodi per la modulazione di splicing di smn2 in un soggetto
EP2446036B1 (en) 2009-06-24 2017-03-01 CuRNA, Inc. Treatment of tumor necrosis factor receptor 2 (tnfr2) related diseases by inhibition of natural antisense transcript to tnfr2
EP2446037B1 (en) 2009-06-26 2016-04-20 CuRNA, Inc. Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene
CN102762731B (zh) 2009-08-05 2018-06-22 库尔纳公司 通过抑制针对胰岛素基因(ins)的天然反义转录物来治疗胰岛素基因(ins)相关的疾病
EP2810643A3 (en) 2009-08-14 2015-03-11 Alnylam Pharmaceuticals Inc. Lipid formulated compositions and mehods for inhibiting expression of a gene from the ebola virus
JP5964232B2 (ja) 2009-08-25 2016-08-03 カッパーアールエヌエー，インコーポレイテッド ‘ｉｑモチーフ含有ｇｔｐアーゼ活性化タンパク質’（ｉｑｇａｐ）に対する天然アンチセンス転写産物の阻害によるｉｑｇａｐ関連疾患の治療
AU2010289400B2 (en) 2009-09-02 2014-10-23 Curis, Inc. Mutant smoothened and methods of using the same
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
US9376690B2 (en) 2009-10-30 2016-06-28 Northwestern University Templated nanoconjugates
EP3002297B1 (en) 2009-11-30 2020-04-08 F. Hoffmann-La Roche AG Antibodies for treating and diagnosing tumors expressing slc34a2 (tat211)
US9687550B2 (en) 2009-12-07 2017-06-27 Arbutus Biopharma Corporation Compositions for nucleic acid delivery
CA2782366A1 (en) 2009-12-16 2011-07-14 Opko Curna, Llc Treatment of membrane bound transcription factor peptidase, site 1 (mbtps1) related diseases by inhibition of natural antisense transcript to mbtps1
WO2011075656A1 (en) 2009-12-18 2011-06-23 The University Of British Columbia Methods and compositions for delivery of nucleic acids
EP2515947B1 (en) 2009-12-23 2021-10-06 CuRNA, Inc. Treatment of uncoupling protein 2 (ucp2) related diseases by inhibition of natural antisense transcript to ucp2
CA2782373C (en) 2009-12-23 2019-03-26 Opko Curna, Llc Treatment of hepatocyte growth factor (hgf) related diseases by inhibition of natural antisense transcript to hgf
RU2611186C2 (ru) 2009-12-29 2017-02-21 Курна, Инк. ЛЕЧЕНИЕ ЗАБОЛЕВАНИЙ, СВЯЗАННЫХ С ОПУХОЛЕВЫМ БЕЛКОМ 63 (р63), ПУТЕМ ИНГИБИРОВАНИЯ ПРИРОДНОГО АНТИСМЫСЛОВОГО ТРАНСКРИПТА К р63
ES2657452T3 (es) 2009-12-29 2018-03-05 Curna, Inc. Tratamiento de enfermedades relacionadas con el factor respiratorio nuclear 1 (NRF1) mediante inhibición de transcrito antisentido natural a NRF1
WO2011082409A2 (en) 2010-01-04 2011-07-07 Curna, Inc. Treatment of interferon regulatory factor 8 (irf8) related diseases by inhibition of natural antisense transcript to irf8
KR101853509B1 (ko) 2010-01-06 2018-04-30 큐알엔에이, 인크. 췌장 발달 유전자에 대한 천연 안티센스 전사체의 억제에 의한 췌장 발달 유전자와 관련된 질환의 치료
US9200277B2 (en) 2010-01-11 2015-12-01 Curna, Inc. Treatment of sex hormone binding globulin (SHBG) related diseases by inhibition of natural antisense transcript to SHBG
US8946182B2 (en) 2010-01-25 2015-02-03 Curna, Inc. Treatment of RNASE H1 related diseases by inhibition of natural antisense transcript to RNASE H1
WO2011100131A2 (en) 2010-01-28 2011-08-18 Alnylam Pharmacuticals, Inc. Monomers and oligonucleotides comprising cycloaddition adduct(s)
WO2011094580A2 (en) 2010-01-28 2011-08-04 Alnylam Pharmaceuticals, Inc. Chelated copper for use in the preparation of conjugated oligonucleotides
CA2824843A1 (en) 2010-02-04 2011-08-11 Ico Therapeutics Inc. Dosing regimens for treating and preventing ocular disorders using c-raf antisense
KR101838308B1 (ko) 2010-02-22 2018-03-13 큐알엔에이, 인크. 피롤린-5-카르복실레이트 환원효소 1(ｐｙｃｒ1)에 대한 천연 안티센스 전사체의 억제에 의한 ｐｙｃｒ1과 관련된 질환의 치료
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
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
WO2011105901A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 9 (c9) and uses thereof
JP5988436B2 (ja) 2010-02-23 2016-09-07 ジェネンテック， インコーポレイテッド 腫瘍の診断と治療のための組成物と方法
WO2011112516A1 (en) 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
EP2544703A4 (en) 2010-03-12 2013-09-18 Brigham & Womens Hospital METHODS OF TREATING VASCULAR INFLAMMATORY DISORDERS
EP3329924B1 (en) 2010-03-29 2021-05-05 Alnylam Pharmaceuticals, Inc. Dsrna therapy for transthyretin (ttr) related ocular amyloidosis
WO2011123621A2 (en) 2010-04-01 2011-10-06 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
WO2011133868A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. Conformationally restricted dinucleotide monomers and oligonucleotides
US10913767B2 (en) 2010-04-22 2021-02-09 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
US9725479B2 (en) 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
EP2606057B1 (en) 2010-04-28 2016-06-15 Ionis Pharmaceuticals, Inc. Modified 5' diphosphate nucleosides and oligomeric compounds prepared therefrom
JP6005628B2 (ja) 2010-04-28 2016-10-12 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Ｉｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 修飾ヌクレオシド、その類似体、およびこれらから調製されるオリゴマー化合物
WO2011139699A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
SMT201700209T1 (it) 2010-04-29 2017-05-08 Ionis Pharmaceuticals Inc Modulazione dell’espressione della transtiretina
WO2011139911A2 (en) 2010-04-29 2011-11-10 Isis Pharmaceuticals, Inc. Lipid formulated single stranded rna
WO2011139985A1 (en) 2010-05-03 2011-11-10 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
KR101936011B1 (ko) 2010-05-03 2019-01-07 큐알엔에이, 인크. 시르투인 (sirt)에 대한 자연 안티센스 전사체의 저해에 의한 시르투인 (sirt) 관련된 질환의 치료
TWI531370B (zh) 2010-05-14 2016-05-01 可娜公司 藉由抑制ｐａｒ４天然反股轉錄本治療ｐａｒ４相關疾病
EP3299464B1 (en) 2010-05-26 2019-10-02 CuRNA, Inc. Treatment of atonal homolog 1 (atoh1) related diseases by inhibition of natural antisense transcript to atoh1
CA3102008A1 (en) 2010-06-02 2011-12-08 Alnylam Pharmaceuticals, Inc. Compositions and methods directed to treating liver fibrosis
US9638632B2 (en) 2010-06-11 2017-05-02 Vanderbilt University Multiplexed interferometric detection system and method
US20130236968A1 (en) 2010-06-21 2013-09-12 Alnylam Pharmaceuticals, Inc. Multifunctional copolymers for nucleic acid delivery
WO2012009402A2 (en) 2010-07-14 2012-01-19 Opko Curna Llc Treatment of discs large homolog (dlg) related diseases by inhibition of natural antisense transcript to dlg
WO2012016188A2 (en) 2010-07-30 2012-02-02 Alnylam Pharmaceuticals, Inc. Methods and compositions for delivery of active agents
US20130323269A1 (en) 2010-07-30 2013-12-05 Muthiah Manoharan Methods and compositions for delivery of active agents
WO2012044992A2 (en) 2010-09-30 2012-04-05 Agency For Science, Technology And Research (A*Star) Methods and reagents for detection and treatment of esophageal metaplasia
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
KR101886457B1 (ko) 2010-10-06 2018-08-07 큐알엔에이, 인크. 시알리다아제 4 (neu4)에 대한 자연 안티센스 전사체의 저해에 의한 neu4 관련된 질환의 치료
WO2012048113A2 (en) 2010-10-07 2012-04-12 The General Hospital Corporation Biomarkers of cancer
WO2012054723A2 (en) 2010-10-22 2012-04-26 Opko Curna Llc Treatment of alpha-l-iduronidase (idua) related diseases by inhibition of natural antisense transcript to idua
CN103201387B (zh) 2010-10-27 2018-02-02 库尔纳公司 通过抑制干扰素相关发育调节因子1(ifrd1)的天然反义转录物而治疗ifrd1相关疾病
CN103370054A (zh) 2010-11-09 2013-10-23 阿尔尼拉姆医药品有限公司 用于抑制Eg5和VEGF基因的表达的脂质配制的组合物和方法
CA2817256A1 (en) 2010-11-12 2012-05-18 The General Hospital Corporation Polycomb-associated non-coding rnas
KR102010598B1 (ko) 2010-11-23 2019-08-13 큐알엔에이, 인크. Nanog에 대한 자연 안티센스 전사체의 저해에 의한 nanog 관련된 질환의 치료
US9193973B2 (en) 2010-12-10 2015-11-24 Alynylam Pharmaceuticals, Inc. Compositions and methods for increasing erythropoietin (EPO) production
WO2012079046A2 (en) 2010-12-10 2012-06-14 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of klf-1 and bcl11a genes
JP5902197B2 (ja) 2011-01-11 2016-04-13 アルニラム・ファーマシューティカルズ・インコーポレーテッド Ｐｅｇ化脂質および薬剤送達のためのそれらの使用
EP2663323B1 (en) 2011-01-14 2017-08-16 The General Hospital Corporation Methods targeting mir-128 for regulating cholesterol/lipid metabolism
PL2670411T3 (pl) 2011-02-02 2019-09-30 Excaliard Pharmaceuticals, Inc. Związki antysensowne ukierunkowane na czynnik wzrostu tkanki łącznej (ctgf) do leczenia bliznowców lub blizn przerostowych
EP2678026B1 (en) 2011-02-21 2016-05-18 The University of Zurich Ankyrin g for use in the treatment of neurodegenerative disorders
US9562853B2 (en) 2011-02-22 2017-02-07 Vanderbilt University Nonaqueous backscattering interferometric methods
EP2691121A4 (en) 2011-03-29 2015-08-26 Alnylam Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TMPRSS6 GENE
ES2653247T3 (es) 2011-06-09 2018-02-06 Curna, Inc. Tratamiento de enfermedades relacionadas con la frataxina (FXN) mediante inhibición del transcrito antisentido natural al gen FXN
EP2723758B1 (en) 2011-06-21 2018-06-20 Alnylam Pharmaceuticals, Inc. Angiopoietin-like 3 (angptl3) irna compostions and methods of use thereof
US9068184B2 (en) 2011-06-21 2015-06-30 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein C (PROC) genes
RU2631805C2 (ru) 2011-06-21 2017-09-26 Элнилэм Фармасьютикалз, Инк. Композиции и способы ингибирования экспрессии генов аполипопротеина с-iii (арос3)
WO2012178033A2 (en) 2011-06-23 2012-12-27 Alnylam Pharmaceuticals, Inc. Serpina1 sirnas: compositions of matter and methods of treatment
JP2014526887A (ja) 2011-08-01 2014-10-09 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 造血幹細胞移植の成功率を改善する方法
CA2847698C (en) 2011-09-14 2020-09-01 Northwestern University Nanoconjugates able to cross the blood-brain barrier
US9580708B2 (en) 2011-09-14 2017-02-28 Rana Therapeutics, Inc. Multimeric oligonucleotides compounds
JP6250543B2 (ja) 2011-09-27 2017-12-20 アルニラム・ファーマシューティカルズ・インコーポレーテッド ジ脂肪族置換ｐｅｇ化脂質
EP3170899B1 (en) 2011-10-11 2020-06-24 The Brigham and Women's Hospital, Inc. Microrna mir-155 in neurodegenerative disorders
US10557136B2 (en) 2011-12-12 2020-02-11 Oncolmmunin Inc. In vivo delivery of oligonucleotides
KR20140102759A (ko) 2011-12-16 2014-08-22 모더나 세라퓨틱스, 인코포레이티드 변형된 뉴클레오사이드, 뉴클레오타이드 및 핵산 조성물
RU2661104C2 (ru) 2012-03-15 2018-07-11 КЁРНА, Инк. Лечение заболеваний, связанных с нейтрофическим фактором головного мозга (bdnf), путем ингибирования природного антисмыслового транскрипта bdnf
WO2013151672A2 (en) 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of oncology-related proteins and peptides
CA2868391A1 (en) 2012-04-02 2013-10-10 Stephane Bancel Polynucleotides comprising n1-methyl-pseudouridine and methods for preparing the same
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
CN104704122A (zh) 2012-04-20 2015-06-10 艾珀特玛治疗公司 产热的miRNA调节剂
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
EP2841572B1 (en) 2012-04-27 2019-06-19 Duke University Genetic correction of mutated genes
US9273949B2 (en) 2012-05-11 2016-03-01 Vanderbilt University Backscattering interferometric methods
WO2013173652A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
EA201492123A1 (ru) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии семейства генов smn
US20150216892A1 (en) 2012-08-03 2015-08-06 Aptamir Therapeutics, Inc. Cell-specific delivery of mirna modulators for the treatment of obesity and related disorders
DK2920304T3 (da) 2012-11-15 2019-05-13 Roche Innovation Ct Copenhagen As Oligonukleotidkonjugater
LT2922554T (lt) 2012-11-26 2022-06-27 Modernatx, Inc. Terminaliai modifikuota rnr
US20140200261A1 (en) 2013-01-17 2014-07-17 Moderna Therapeutics, Inc. Signal-sensor polynucleotides for the alteration of cellular phenotypes
DK2951305T3 (en) 2013-01-30 2018-10-29 Hoffmann La Roche LNA oligonucleotide KULHYDRATKONJUGATER
WO2014118272A1 (en) 2013-01-30 2014-08-07 Santaris Pharma A/S Antimir-122 oligonucleotide carbohydrate conjugates
US20150366890A1 (en) 2013-02-25 2015-12-24 Trustees Of Boston University Compositions and methods for treating fungal infections
US20160024181A1 (en) 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
EP2971010B1 (en) 2013-03-14 2020-06-10 ModernaTX, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
IL288931B2 (en) 2013-03-14 2025-05-01 Alnylam Pharmaceuticals Inc Compositions comprising a complementary portion of C5 IRNA and methods of using them
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
US9828582B2 (en) 2013-03-19 2017-11-28 Duke University Compositions and methods for the induction and tuning of gene expression
CA2918194C (en) 2013-03-27 2022-12-06 The General Hospital Corporation Methods and agents for treating alzheimer's disease
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
SG10201804472YA (en) 2013-05-22 2018-07-30 Alnylam Pharmaceuticals Inc SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
CA2918787A1 (en) 2013-06-13 2014-12-18 George Tachas Combination therapy
US9879265B2 (en) 2013-06-27 2018-01-30 Roche Innovation Center Copenhagen A/S Oligonucleotide conjugates
PT3019619T (pt) 2013-07-11 2021-11-11 Modernatx Inc Composições que compreendem polinucleótidos sintéticos que codificam proteínas relacionadas com crispr e sgarn sintéticos e métodos de utilização
LT3041854T (lt) 2013-08-08 2020-03-25 The Scripps Research Institute Nukleorūgščių specifinės vietos žymėjimo fermentais in vitro būdas, įvedant nenatūralius nukleotidus
WO2015034925A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Circular polynucleotides
US20160194625A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Chimeric polynucleotides
WO2015050990A1 (en) 2013-10-02 2015-04-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
SG11201602503TA (en) 2013-10-03 2016-04-28 Moderna Therapeutics Inc Polynucleotides encoding low density lipoprotein receptor
PT3052628T (pt) 2013-10-04 2020-06-01 Alnylam Pharmaceuticals Inc Composições e métodos para inibição da expressão do gene alas1
AU2014351482B2 (en) 2013-11-22 2020-08-20 Mina Therapeutics Limited C/EBP alpha short activating RNA compositions and methods of use
CA2844640A1 (en) 2013-12-06 2015-06-06 The University Of British Columbia Method for treatment of castration-resistant prostate cancer
SG11201604692UA (en) 2013-12-12 2016-07-28 Alnylam Pharmaceuticals Inc Complement component irna compositions and methods of use thereof
CA2937539A1 (en) 2014-02-04 2015-08-13 Genentech, Inc. Mutant smoothened and methods of using the same
CN106103718B (zh) 2014-02-11 2021-04-02 阿尔尼拉姆医药品有限公司 己酮糖激酶(KHK)iRNA组合物及其使用方法
ES2891738T3 (es) 2014-04-09 2022-01-31 Scripps Research Inst Incorporación de nucleósidos trifosfato no naturales o modificados a células a través de transportadores de ácido nucleico trifosfato
US20170044538A1 (en) 2014-04-17 2017-02-16 Biogen Ma Inc. Compositions and Methods for Modulation of SMN2 Splicing in a Subject
WO2015171918A2 (en) 2014-05-07 2015-11-12 Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
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
GB201408623D0 (en) 2014-05-15 2014-07-02 Santaris Pharma As Oligomers and oligomer conjugates
EA201692370A1 (ru) 2014-05-22 2017-03-31 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ иРНК АНГИОТЕНЗИНОГЕНА (AGT) И СПОСОБЫ ИХ ИСПОЛЬЗОВАНИЯ
EP3164113B1 (en) 2014-06-04 2019-03-27 Exicure, Inc. Multivalent delivery of immune modulators by liposomal spherical nucleic acids for prophylactic or therapeutic applications
ES2908962T3 (es) 2014-06-10 2022-05-04 Univ Erasmus Med Ct Rotterdam Oligonucleótidos antisentido útiles en el tratamiento de la enfermedad de Pompe
EP3161159B1 (en) 2014-06-25 2020-08-05 The General Hospital Corporation Targeting human satellite ii (hsatii)
CA2955250A1 (en) 2014-07-16 2016-01-21 Moderna Therapeutics, Inc. Chimeric polynucleotides
EP3171895A1 (en) 2014-07-23 2017-05-31 Modernatx, Inc. Modified polynucleotides for the production of intrabodies
IL250302B (en) 2014-07-31 2022-07-01 Uab Research Foundation Apoe mimetic peptides and higher potency to clear plasma cholesterol
WO2016033472A1 (en) 2014-08-29 2016-03-03 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
US10556020B2 (en) 2014-09-26 2020-02-11 University Of Massachusetts RNA-modulating agents
EP3207138B1 (en) 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
EP3212794B1 (en) 2014-10-30 2021-04-07 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
AU2015349680A1 (en) 2014-11-21 2017-06-08 Northwestern University The sequence-specific cellular uptake of spherical nucleic acid nanoparticle conjugates
EP4088741A1 (en) 2014-12-08 2022-11-16 The Board of Regents of the University of Texas System Lipocationic polymers and uses thereof
AU2015364508A1 (en) 2014-12-18 2017-07-06 Alnylam Pharmaceuticals, Inc. ReversirTM compounds
JP2018506715A (ja) 2015-01-23 2018-03-08 ヴァンダービルト ユニバーシティー 堅固なインターフェロメーター及びその使用方法
EP3256487A4 (en) 2015-02-09 2018-07-18 Duke University Compositions and methods for epigenome editing
JP2018510621A (ja) 2015-02-13 2018-04-19 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． パタチン様ホスホリパーゼドメイン含有３（ＰＮＰＬＡ３）ｉＲＮＡ組成物およびその使用方法
EP3262173A2 (en) 2015-02-23 2018-01-03 Crispr Therapeutics AG Materials and methods for treatment of human genetic diseases including hemoglobinopathies
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.
EP4365291A3 (en) 2015-06-12 2024-08-14 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
EP3310918B1 (en) 2015-06-18 2020-08-05 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, hao1) and methods of use thereof
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
WO2016210241A1 (en) 2015-06-26 2016-12-29 Beth Israel Deaconess Medical Center, Inc. Cancer therapy targeting tetraspanin 33 (tspan33) in myeloid derived suppressor cells
WO2017004243A1 (en) 2015-06-29 2017-01-05 Caris Science, Inc. Therapeutic oligonucleotides
US10494632B2 (en) 2015-07-10 2019-12-03 Alnylam Pharmaceuticals, Inc. Insulin-like growth factor binding protein, acid labile subunit (IGFALS) compositions and methods of use thereof
DK3324980T3 (da) 2015-07-17 2022-02-14 Alnylam Pharmaceuticals Inc Multimålrettede enkeltenhedskonjugater
AU2016298317B2 (en) 2015-07-28 2021-02-18 Caris Science, Inc. Targeted oligonucleotides
US10889813B2 (en) 2015-09-02 2021-01-12 Alnylam Pharmaceuticals, Inc. Programmed cell death 1 ligand 1 (PD-L1) iRNA compositions and methods of use thereof
EP3950003A1 (en) 2015-09-14 2022-02-09 The Board of Regents of the University of Texas System Lipocationic dendrimers and uses thereof
AU2016339053A1 (en) 2015-09-24 2018-04-12 Crispr Therapeutics Ag Novel family of RNA-programmable endonucleases and their uses in genome editing and other applications
EP3362571A4 (en) 2015-10-13 2019-07-10 Duke University GENOM ENGINEERING WITH TYPE I CRISPR SYSTEMS IN EUKARYOTIC CELLS
CN108513546A (zh) 2015-10-28 2018-09-07 克里斯珀医疗股份公司 用于治疗杜氏肌营养不良症的材料和方法
EP3370734B1 (en) 2015-11-05 2023-01-04 Children's Hospital Los Angeles Antisense oligo for use in treating acute myeloid leukemia
BR112018008971A2 (pt) 2015-11-06 2018-11-27 Crispr Therapeutics Ag materiais e métodos para tratamento de doença de armazenamento de glicogênio tipo 1a
CA3005878A1 (en) 2015-11-19 2017-05-26 The Brigham And Women's Hospital, Inc. Lymphocyte antigen cd5-like (cd5l)-interleukin 12b (p40) heterodimers in immunity
US12214054B2 (en) 2015-11-30 2025-02-04 Duke University Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
EP3967758A1 (en) 2015-12-01 2022-03-16 CRISPR Therapeutics AG Materials and methods for treatment of alpha-1 antitrypsin deficiency
TW202423457A (zh) 2015-12-07 2024-06-16 美商健贊公司 治療ｓｅｒｐｉｎｃ１相關疾患之方法及組成物
EP3387127A1 (en) 2015-12-07 2018-10-17 Erasmus University Medical Center Rotterdam Enzymatic replacement therapy and antisense therapy for pompe disease
US11761007B2 (en) 2015-12-18 2023-09-19 The Scripps Research Institute Production of unnatural nucleotides using a CRISPR/Cas9 system
KR20180118111A (ko) 2015-12-23 2018-10-30 크리스퍼 테라퓨틱스 아게 근위축성 측색 경화증 및/또는 전두측두엽 퇴행의 치료 물질 및 방법
WO2017132483A1 (en) 2016-01-29 2017-08-03 Vanderbilt University Free-solution response function interferometry
WO2017134529A1 (en) 2016-02-02 2017-08-10 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
EP3416689B1 (en) 2016-02-18 2023-01-18 CRISPR Therapeutics AG Materials and methods for treatment of severe combined immunodeficiency (scid) or omenn syndrome
ES2999146T3 (en) 2016-02-25 2025-02-24 Brigham & Womens Hospital Inc Treatment methods for fibrosis targeting smoc2
JP6748219B2 (ja) 2016-03-14 2020-08-26 エフ．ホフマン−ラ ロシュ アーゲーＦ． Ｈｏｆｆｍａｎｎ−Ｌａ Ｒｏｃｈｅ Ａｋｔｉｅｎｇｅｓｅｌｌｓｃｈａｆｔ Ｐｄ−ｌ１発現低減用のオリゴヌクレオチド
EP3429632B1 (en) 2016-03-16 2023-01-04 CRISPR Therapeutics AG Materials and methods for treatment of hereditary haemochromatosis
EP3828272B9 (en) 2016-03-18 2023-12-27 Caris Science, Inc. Method of enrichment of oligonucleotide probes
SG11201809144XA (en) 2016-04-18 2018-11-29 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2017191503A1 (en) 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
WO2017201076A1 (en) 2016-05-16 2017-11-23 The Board Of Regents Of The University Of Texas System Cationic sulfonamide amino lipids and amphiphilic zwitterionic amino lipids
AU2017271579B2 (en) 2016-05-25 2023-10-19 Caris Science, Inc. Oligonucleotide probes and uses thereof
JP2019518028A (ja) 2016-06-10 2019-06-27 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Ａｌｎｙｌａｍ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ， Ｉｎｃ． 補体成分Ｃ５ｉＲＮＡ組成物及び発作性夜間血色素尿症（ＰＮＨ）を処置するためのその使用方法
EP3475295B1 (en) 2016-06-24 2022-08-10 The Scripps Research Institute Novel nucleoside triphosphate transporter and uses thereof
US11427838B2 (en) 2016-06-29 2022-08-30 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of myotonic dystrophy type 1 (DM1) and other related disorders
ES2989940T3 (es) 2016-06-29 2024-11-28 Crispr Therapeutics Ag Materiales y métodos para el tratamiento de la ataxia de Friedreich y otros trastornos relacionados
US11174469B2 (en) 2016-06-29 2021-11-16 Crispr Therapeutics Ag Materials and methods for treatment of Amyotrophic Lateral Sclerosis (ALS) and other related disorders
US11459587B2 (en) 2016-07-06 2022-10-04 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of pain related disorders
WO2018007976A1 (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
CA3030701A1 (en) 2016-07-11 2018-01-18 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
JP7490211B2 (ja) 2016-07-19 2024-05-27 デューク ユニバーシティ Ｃｐｆ１に基づくゲノム編集の治療適用
WO2018020323A2 (en) 2016-07-25 2018-02-01 Crispr Therapeutics Ag Materials and methods for treatment of fatty acid disorders
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
NL2017295B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Antisense oligomeric compound for Pompe disease
EP3532638A4 (en) 2016-10-31 2020-07-29 University of Massachusetts TARGETING MICROARN-101-3 P IN CARCINOTHERAPY
TW202313978A (zh) 2016-11-23 2023-04-01 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
WO2018111834A1 (en) 2016-12-13 2018-06-21 Seattle Children's Hospital (dba Seattle Children's Research Institute) Methods of exogenous drug activation of chemical-induced signaling complexes expressed in engineered cells in vitro and in vivo
AU2017376950B2 (en) 2016-12-16 2024-02-22 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing TTR-associated diseases using transthyretin (TTR) iRNA compositions
WO2018136758A1 (en) 2017-01-23 2018-07-26 Regeneron Pharmaceuticals, Inc. 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
WO2018154418A1 (en) 2017-02-22 2018-08-30 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
US11559588B2 (en) 2017-02-22 2023-01-24 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
CA3054031A1 (en) 2017-02-22 2018-08-30 Crispr Therapeutics Ag Compositions and methods for gene editing
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
IL269927B2 (en) 2017-04-18 2025-04-01 Alnylam Pharmaceuticals Inc Methods of treating patients with hepatitis B virus infection
US12208140B2 (en) 2017-04-21 2025-01-28 The Broad Institute, Inc. Targeted delivery to beta cells
US11622977B2 (en) 2017-05-12 2023-04-11 Crispr Therapeutics Ag Materials and methods for engineering cells and uses thereof in immuno-oncology
CA3069150A1 (en) 2017-07-10 2019-01-17 Genzyme Corporation Methods and compositions for treating a bleeding event in a subject having hemophilia
IL271903B2 (en) 2017-07-11 2025-04-01 Synthorx Inc Combination of unnatural nucleotides and methods therefor
CA3069868A1 (en) 2017-07-13 2019-01-17 Alnylam Pharmaceuticals Inc. Lactate dehydrogenase a (ldha) irna compositions and methods of use thereof
EP3661955A4 (en) 2017-08-03 2021-05-26 Synthorx, Inc. CYTOKINE CONJUGATES FOR THE TREATMENT OF AUTOIMMUNE DISEASES
US11162099B2 (en) 2017-09-08 2021-11-02 Mina Therapeutics Limited HNF4A saRNA compositions and methods of use
EP3679140B1 (en) 2017-09-08 2022-11-16 MiNA Therapeutics Limited Stabilized cebpa sarna compositions and methods of use
WO2019055460A1 (en) 2017-09-13 2019-03-21 The Children's Medical Center Corporation COMPOSITIONS AND METHODS FOR THE TREATMENT OF TRANSPOSON-ASSOCIATED DISEASES
CN111684070A (zh) 2017-10-17 2020-09-18 克里斯珀医疗股份公司 用于a型血友病基因编辑的组合物和方法
US20210180091A1 (en) 2017-10-26 2021-06-17 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
US11866701B2 (en) 2017-11-01 2024-01-09 Alnylam Pharmaceuticals, Inc. Complement component C3 iRNA compositions and methods of use thereof
US20210363521A1 (en) 2017-11-09 2021-11-25 Vertex Pharmaceuticals Incorporated CRISPR/CAS Systems For Treatment of DMD
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
EP3714055A1 (en) 2017-11-21 2020-09-30 CRISPR Therapeutics AG Materials and methods for treatment of autosomal dominant retinitis pigmentosa
JP2021505587A (ja) 2017-12-05 2021-02-18 バーテックス ファーマシューティカルズ インコーポレイテッドＶｅｒｔｅｘ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ Ｉｎｃｏｒｐｏｒａｔｅｄ Ｃｒｉｓｐｒ−ｃａｓ９改変ｃｄ３４＋ヒト造血幹・前駆細胞及びその使用
EP3724332A1 (en) 2017-12-14 2020-10-21 CRISPR Therapeutics AG Novel rna-programmable endonuclease systems and their use in genome editing and other applications
KR20200110655A (ko) 2017-12-18 2020-09-24 알닐람 파마슈티칼스 인코포레이티드 고이동성 그룹 박스-1 (hmgb1) irna 조성물 및 이의 사용 방법
EP3728595A1 (en) 2017-12-21 2020-10-28 CRISPR Therapeutics AG Materials and methods for treatment of usher syndrome type 2a and/or non-syndromic autosomal recessive retinitis pigmentosa (arrp)
AU2018393050A1 (en) 2017-12-21 2020-06-18 Bayer Healthcare Llc Materials and methods for treatment of Usher Syndrome Type 2A
CN118879592A (zh) 2017-12-29 2024-11-01 斯克利普斯研究所 非天然碱基对组合物及使用方法
EP3737424A4 (en) 2018-01-10 2021-10-27 Translate Bio MA, Inc. COMPOSITIONS AND METHODS FOR FACILITATING THE DELIVERY OF SYNTHETIC NUCLEIC ACIDS TO CELLS
MA51637A (fr) 2018-01-12 2020-11-18 Bayer Healthcare Llc Compositions et méthodes pour l'édition génique par ciblage de la transferrine
US20190233816A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
EP3749767A1 (en) 2018-02-05 2020-12-16 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
US11268077B2 (en) 2018-02-05 2022-03-08 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
MA51869A (fr) 2018-02-16 2020-12-23 Bayer Healthcare Llc Compositions et méthodes pour l'édition génique par ciblage du fibrinogène-alpha
ES3010583T3 (en) 2018-02-26 2025-04-03 Synthorx Inc Il-15 conjugates and uses thereof
JP7550648B2 (ja) 2018-03-19 2024-09-13 クリスパー セラピューティクス アーゲー 新規ｒｎａプログラム可能エンドヌクレアーゼ系およびその使用
US12049631B2 (en) 2018-03-30 2024-07-30 Rheinische Friedrich-Wilhelms-Universitat Bonn Aptamers for targeted activation of T cell-mediated immunity
KR20200141470A (ko) 2018-04-06 2020-12-18 칠드런'즈 메디컬 센터 코포레이션 체세포 재프로그래밍 및 각인의 조정을 위한 조성물 및 방법
US11566246B2 (en) 2018-04-12 2023-01-31 Mina Therapeutics Limited SIRT1-saRNA compositions and methods of use
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
CA3091490A1 (en) 2018-04-27 2019-10-31 Seattle Children's Hospital (dba Seattle Children's Research Institute) Rapamycin resistant cells
TW202016301A (zh) 2018-05-07 2020-05-01 美商阿里拉姆製藥股份有限公司 肝外遞送技術
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
AU2019321375A1 (en) 2018-08-13 2021-03-11 Alnylam Pharmaceuticals, Inc. Hepatitis B virus (HBV) dsRNA agent compositions and methods of use thereof
EP3837367A1 (en) 2018-08-16 2021-06-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
US12281308B2 (en) 2018-08-29 2025-04-22 University Of Massachusetts Inhibition of protein kinases to treat Friedreich ataxia
EP3853364A1 (en) 2018-09-18 2021-07-28 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
CA3114396A1 (en) 2018-09-28 2020-04-02 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
AU2019362000A1 (en) 2018-10-17 2021-05-20 Bayer Healthcare Llc Compositions and methods for delivering 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
CN113710799B (zh) 2018-11-28 2024-11-12 克里斯珀医疗股份公司 用于在LNP中使用的编码CAS9的优化mRNA
CA3123617A1 (en) 2018-12-20 2020-06-25 Praxis Precision Medicines, Inc. Compositions and methods for the treatment of kcnt1 related disorders
CN113543791B (zh) 2018-12-20 2025-08-05 维尔生物科技有限公司 组合hbv疗法
CN113557023A (zh) 2019-01-16 2021-10-26 建新公司 Serpinc1 iRNA组合物及其使用方法
CN113660946A (zh) 2019-02-06 2021-11-16 新索思股份有限公司 Il-2缀合物及其使用方法
AU2020221340A1 (en) 2019-02-15 2021-09-16 Bayer Healthcare Llc Gene editing for hemophilia A with improved Factor VIII expression
WO2020171889A1 (en) 2019-02-19 2020-08-27 University Of Rochester Blocking lipid accumulation or inflammation in thyroid eye disease
MA55297A (fr) 2019-03-12 2022-01-19 Bayer Healthcare Llc Nouveaux systèmes d'endonucléase à arn programmable haute fidélité et leurs utilisations
US20220211740A1 (en) 2019-04-12 2022-07-07 Mina Therapeutics Limited Sirt1-sarna compositions and methods of use
PH12021552870A1 (en) 2019-05-13 2022-03-21 Vir Biotechnology Inc Compositions and methods for treating hepatitis b virus (hbv) infection
AU2020279101B2 (en) 2019-05-17 2025-07-24 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
JP7728181B2 (ja) 2019-06-14 2025-08-22 ザ スクリプス リサーチ インスティテュート 半合成生物における複製、転写および翻訳のための試薬ならびに方法
EP4007812A1 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Serpin family f member 2 (serpinf2) irna compositions and methods of use thereof
EP4007811A2 (en) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Carboxypeptidase b2 (cpb2) irna compositions and methods of use thereof
WO2021030522A1 (en) 2019-08-13 2021-02-18 Alnylam Pharmaceuticals, Inc. SMALL RIBOSOMAL PROTEIN SUBUNIT 25 (RPS25) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
BR112022002442A2 (pt) 2019-08-15 2022-07-05 Synthorx Inc Terapias de imuno-oncologia com conjugados de il-2
WO2021032777A1 (en) 2019-08-19 2021-02-25 Mina Therapeutics Limited Oligonucleotide conjugate compositions and methods of use
TW202122401A (zh) 2019-08-23 2021-06-16 美商欣爍克斯公司 新穎之ｉｌ－１５接合物及其用途
BR112022003860A2 (pt) 2019-09-03 2022-08-16 Alnylam Pharmaceuticals Inc Composições e métodos para inibir a expressão do gene lect2
CN114746122A (zh) 2019-09-10 2022-07-12 新索思股份有限公司 Il-2缀合物和治疗自身免疫性疾病的使用方法
US20220389429A1 (en) 2019-10-04 2022-12-08 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
MX2022004726A (es) 2019-10-22 2022-05-13 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de componente c3 de complemento y metodos de uso de las mismas.
US20230040920A1 (en) 2019-11-01 2023-02-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajb1-prkaca fusion gene expression
KR20220094213A (ko) 2019-11-01 2022-07-05 알닐람 파마슈티칼스 인코포레이티드 헌팅틴 (HTT) iRNA 제제 조성물 및 이의 사용 방법
AU2020380275A1 (en) 2019-11-04 2022-04-14 Synthorx, Inc. Interleukin 10 conjugates and uses thereof
CA3160329A1 (en) 2019-11-06 2021-05-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
EP4055165A1 (en) 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr) irna compositions and methods of use thereof for treating or preventing ttr-associated ocular diseases
PH12022551158A1 (en) 2019-11-13 2023-10-02 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
IL293824A (en) 2019-12-13 2022-08-01 Alnylam Pharmaceuticals Inc Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
TW202138559A (zh) 2019-12-16 2021-10-16 美商阿尼拉製藥公司 含類ＰＡＴＡＴＩＮ磷脂酶結構域３（ＰＮＰＬＡ３）ｉＲＮＡ組成物及其使用方法
US20230054569A1 (en) 2019-12-18 2023-02-23 Alia Therapeutics Srl Compositions and methods for treating retinitis pigmentosa
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)
EP4103714A1 (en) 2020-02-10 2022-12-21 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing vegf-a expression
KR20220143106A (ko) 2020-02-18 2022-10-24 알닐람 파마슈티칼스 인코포레이티드 아포지질단백질 C3 (APOC3) iRNA 조성물 및 이의 사용 방법
PH12022552327A1 (en) 2020-03-04 2023-11-29 Verve Therapeutics Inc Compositions and methods for targeted rna delivery
EP4114947A1 (en) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Complement component c3 irna compositions and methods of use thereof for treating or preventing complement component c3-associated diseases
CA3174725A1 (en) 2020-03-06 2021-09-10 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
IL296106A (en) 2020-03-06 2022-11-01 Alnylam Pharmaceuticals Inc Compositions and methods for inhibiting expression of transthyretin (ttr)
WO2021188611A1 (en) 2020-03-18 2021-09-23 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating subjects having a heterozygous alanine-glyoxylate aminotransferase gene (agxt) variant
CN116209759A (zh) 2020-03-26 2023-06-02 阿尔尼拉姆医药品有限公司 冠状病毒iRNA组合物及其使用方法
EP4127171A2 (en) 2020-03-30 2023-02-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing dnajc15 gene expression
AR121769A1 (es) 2020-04-06 2022-07-06 Alnylam Pharmaceuticals Inc Composiciones y métodos para el silenciamiento de la expresión de myoc
WO2021206917A1 (en) 2020-04-07 2021-10-14 Alnylam Pharmaceuticals, Inc. ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4133077A1 (en) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Transmembrane serine protease 2 (tmprss2) irna compositions and methods of use thereof
US20230159933A1 (en) 2020-04-07 2023-05-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing scn9a expression
BR112022021813A2 (pt) 2020-04-27 2023-01-17 Alnylam Pharmaceuticals Inc Composições de agente de apolipoproteína e (apoe) irna e métodos de uso das mesmas
CA3181198A1 (en) 2020-04-30 2021-11-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
EP4150090A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
EP4150078A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate lyase (asl)
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)
EP4150087A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of gap junction protein beta 2 (gjb2)
EP4150089A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rs1)
EP4150086A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of leucine rich repeat kinase 2 (lrrk2)
EP4150076A1 (en) 2020-05-15 2023-03-22 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of methyl-cpg binding protein 2 (mecp2)
WO2021231675A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
WO2021237097A1 (en) 2020-05-21 2021-11-25 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting marc1 gene expression
CN115885042A (zh) 2020-05-22 2023-03-31 波涛生命科学有限公司 双链寡核苷酸组合物及其相关方法
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
EP4162050A1 (en) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai compositions and methods of use thereof for delivery by inhalation
CA3184289A1 (en) 2020-06-18 2021-12-23 Alnylam Pharmaceuticals, Inc. Xanthine dehydrogenase (xdh) irna compositions and methods of use thereof
EP4171747A1 (en) 2020-06-24 2023-05-03 VIR Biotechnology, Inc. Engineered hepatitis b virus neutralizing antibodies and uses thereof
TW202216203A (zh) 2020-06-25 2022-05-01 美商欣爍克斯公司 Il-2接合物及抗egfr抗體之免疫腫瘤學組合治療
WO2022011214A1 (en) 2020-07-10 2022-01-13 Alnylam Pharmaceuticals, Inc. Circular sirnas
CA3177613A1 (en) 2020-07-10 2022-04-13 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and compositions for treating epilepsy
EP4208549A4 (en) 2020-09-04 2025-04-16 Verve Therapeutics, Inc. Compositions and methods for capping RNAs
WO2022066847A1 (en) 2020-09-24 2022-03-31 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
TW202229552A (zh) 2020-10-05 2022-08-01 美商艾拉倫製藥股份有限公司 Ｇ蛋白－偶合受體７５（ＧＰＲ７５）ｉＲＮＡ組成物及其使用方法
JP2023546009A (ja) 2020-10-09 2023-11-01 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ペムブロリズマブを用いる免疫腫瘍学併用療法
JP2023546010A (ja) 2020-10-09 2023-11-01 シンソークス， インコーポレイテッド Ｉｌ－２コンジュゲートを用いた免疫腫瘍療法
EP4232582A1 (en) 2020-10-23 2023-08-30 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
JP2023547615A (ja) 2020-10-23 2023-11-13 ザ スクリプス リサーチ インスティテュート 非天然ヌクレオチドを含むポリヌクレオチドの逆転写
MX2023005490A (es) 2020-11-13 2023-05-23 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) del factor de coagulacion v (f5) y sus metodos de uso.
EP4259795A1 (en) 2020-12-08 2023-10-18 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
CR20230308A (es) 2020-12-11 2023-09-08 Civi Biopharma Inc Entrega oral de conjugados antisentido que tienen por blanco a pcsk9
WO2022147214A2 (en) 2020-12-31 2022-07-07 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
EP4271695A2 (en) 2020-12-31 2023-11-08 Alnylam Pharmaceuticals, Inc. 2'-modified nucleoside based oligonucleotide prodrugs
EP4274896A1 (en) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Complement component 9 (c9) irna compositions and methods of use thereof
TW202305131A (zh) 2021-02-12 2023-02-01 美商艾拉倫製藥股份有限公司 用於治療或預防超氧歧化酶１－（ＳＯＤ１－）相關的神經退化疾病的超氧歧化酶１（ＳＯＤ１）ｉＲＮＡ組成物及其使用方法
WO2022174101A1 (en) 2021-02-12 2022-08-18 Synthorx, Inc. Skin cancer combination therapy with il-2 conjugates and cemiplimab
EP4291243A1 (en) 2021-02-12 2023-12-20 Synthorx, Inc. Lung cancer combination therapy with il-2 conjugates and an anti-pd-1 antibody or antigen-binding fragment thereof
EP4298220A1 (en) 2021-02-25 2024-01-03 Alnylam Pharmaceuticals, Inc. Prion protein (prnp) irna compositions and methods of use thereof
MX2023009704A (es) 2021-02-26 2023-10-23 Alnylam Pharmaceuticals Inc Composiciones de arni de cetohexocinasa (khk) y métodos de uso de las mismas.
JP2024508896A (ja) 2021-03-04 2024-02-28 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 アンジオポエチン様３（ＡＮＧＰＴＬ３）ｉＲＮＡ組成物およびその使用方法
EP4305169A1 (en) 2021-03-12 2024-01-17 Alnylam Pharmaceuticals, Inc. Glycogen synthase kinase 3 alpha (gsk3a) irna compositions and methods of use thereof
TW202305133A (zh) 2021-03-26 2023-02-01 英商米納治療有限公司 Tmem173 sarna組合物及其使用方法
WO2022212231A2 (en) 2021-03-29 2022-10-06 Alnylam Pharmaceuticals, Inc. Huntingtin (htt) irna agent compositions and methods of use thereof
EP4314293A1 (en) 2021-04-01 2024-02-07 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
CA3214540A1 (en) 2021-04-22 2022-10-27 Pasi A. Janne Compositions and methods for treating cancer
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
JP2024519293A (ja) 2021-04-29 2024-05-10 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 シグナル伝達兼転写活性化因子６（ＳＴＡＴ６）ｉＲＮＡ組成物およびその使用方法
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
JP2024522996A (ja) 2021-06-02 2024-06-25 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 パタチン様ホスホリパーゼドメイン含有３（ＰＮＰＬＡ３）ｉＲＮＡ組成物およびその使用方法
TW202313679A (zh) 2021-06-03 2023-04-01 美商欣爍克斯公司 包含il-2接合物及pd-1拮抗劑之頭頸癌組合療法
CA3221245A1 (en) 2021-06-04 2022-12-08 Alnylam Pharmaceuticals, Inc. Human chromosome 9 open reading frame 72 (c9orf72) irna agent compositions and methods of use thereof
AR126070A1 (es) 2021-06-08 2023-09-06 Alnylam Pharmaceuticals Inc Composiciones y métodos para tratar o prevenir la enfermedad de stargardt y/o trastornos asociados con la proteína transportadora de retinol 4 (rbp4)
EP4101928A1 (en) 2021-06-11 2022-12-14 Bayer AG Type v rna programmable endonuclease systems
IL308896A (en) 2021-06-11 2024-01-01 Bayer Ag Type v rna programmable endonuclease systems
US20250034564A1 (en) 2021-06-29 2025-01-30 Korro Bio, Inc. Methods and Compositions for ADAR-Mediated Editing
US20230194709A9 (en) 2021-06-29 2023-06-22 Seagate Technology Llc Range information detection using coherent pulse sets with selected waveform characteristics
WO2023278576A1 (en) 2021-06-30 2023-01-05 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating an angiotensinogen- (agt-) associated disorder
JP2024527584A (ja) 2021-07-09 2024-07-25 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 ＣＮＳ送達のためのＢｉｓ－ＲＮＡｉ化合物
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
WO2023003922A1 (en) 2021-07-21 2023-01-26 Alnylam Pharmaceuticals, Inc. Metabolic disorder-associated target gene irna compositions and methods of use thereof
KR20240037293A (ko) 2021-07-23 2024-03-21 알닐람 파마슈티칼스 인코포레이티드 베타-카테닌(CTNNB1) iRNA 조성물 및 이의 사용 방법
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
MX2024001194A (es) 2021-08-03 2024-02-27 Alnylam Pharmaceuticals Inc Composiciones de acido ribonucleico de interferencia (arni) de transtiretina (ttr) y sus metodos de uso.
KR20240042016A (ko) 2021-08-04 2024-04-01 알닐람 파마슈티칼스 인코포레이티드 안지오텐시노겐(AGT)을 침묵화하기 위한 iRNA 조성물 및 방법
WO2023019246A1 (en) 2021-08-13 2023-02-16 Alnylam Pharmaceuticals, Inc. Factor xii (f12) irna compositions and methods of use thereof
CA3230404A1 (en) 2021-08-31 2023-03-09 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)
IL311454A (en) 2021-09-20 2024-05-01 Alnylam Pharmaceuticals Inc Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
AU2022364838A1 (en) 2021-10-15 2024-04-11 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
EP4419683A1 (en) 2021-10-22 2024-08-28 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
CA3234636A1 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
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
WO2023099884A1 (en) 2021-12-01 2023-06-08 Mina Therapeutics Limited Pax6 sarna compositions and methods of use
EP4452327A1 (en) 2021-12-20 2024-10-30 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
EP4453196A1 (en) 2021-12-21 2024-10-30 Alia Therapeutics Srl Type ii cas proteins and applications thereof
WO2023122750A1 (en) 2021-12-23 2023-06-29 Synthorx, Inc. Cancer combination therapy with il-2 conjugates and cetuximab
US20250084391A1 (en) 2021-12-23 2025-03-13 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
AU2023227443A1 (en) 2022-03-01 2024-10-10 Crispr Therapeutics Ag Methods and compositions for treating angiopoietin-like 3 (angptl3) related conditions
WO2023170435A1 (en) 2022-03-07 2023-09-14 Mina Therapeutics Limited Il10 sarna compositions and methods of use
WO2023194359A1 (en) 2022-04-04 2023-10-12 Alia Therapeutics Srl Compositions and methods for treatment of usher syndrome type 2a
EP4522742A2 (en) 2022-05-13 2025-03-19 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
AU2023284048A1 (en) 2022-06-10 2024-11-14 Bayer Aktiengesellschaft Novel small type v rna programmable endonuclease systems
EP4547683A2 (en) 2022-06-30 2025-05-07 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
WO2024039776A2 (en) 2022-08-18 2024-02-22 Alnylam Pharmaceuticals, Inc. Universal non-targeting sirna compositions and methods of use thereof
CN119907856A (zh) 2022-09-15 2025-04-29 阿尔尼拉姆医药品有限公司 第13型17β-羟基类固醇脱氢酶(HSD17B13)iRNA组合物及其使用方法
WO2024056880A2 (en) 2022-09-16 2024-03-21 Alia Therapeutics Srl Enqp type ii cas proteins and applications thereof
EP4594492A1 (en) 2022-09-30 2025-08-06 Alnylam Pharmaceuticals, Inc. Modified double-stranded rna agents
WO2024105162A1 (en) 2022-11-16 2024-05-23 Alia Therapeutics Srl Type ii cas proteins and applications thereof
WO2024129743A2 (en) 2022-12-13 2024-06-20 Bluerock Therapeutics Lp 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
WO2024134199A1 (en) 2022-12-22 2024-06-27 Mina Therapeutics Limited Chemically modified sarna compositions and methods of use
WO2024149810A2 (en) 2023-01-11 2024-07-18 Alia Therapeutics Srl Type ii cas proteins and applications thereof
WO2024168010A2 (en) 2023-02-09 2024-08-15 Alnylam Pharmaceuticals, Inc. Reversir molecules and methods of use 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
WO2024216155A1 (en) 2023-04-12 2024-10-17 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery of double-stranded rna agents
WO2024238385A2 (en) 2023-05-12 2024-11-21 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
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
WO2025034422A1 (en) 2023-08-04 2025-02-13 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating ctnnb1-associated disorders
WO2025064660A2 (en) 2023-09-21 2025-03-27 Alnylam Pharmaceuticals, Inc. Activin a receptor type 1c (acvr1c) irna compositions and methods of use thereof
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
WO2025158385A1 (en) 2024-01-25 2025-07-31 Genzyme Corporation Pegylated il-2 for suppressing adaptive immune response to gene therapy

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US3873521A (en) * 1970-09-17 1975-03-25 Astra Laekemedel Ab Esters of {60 -amino penicillins
US4024249A (en) * 1973-03-30 1977-05-17 Ciba-Geigy Corporation Heteroarylacetamido cephalosporin
US4401796A (en) * 1981-04-30 1983-08-30 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
JPS5927900A (ja) * 1982-08-09 1984-02-14 Wakunaga Seiyaku Kk 固定化オリゴヌクレオチド
JPH106696A (ja) * 1996-06-19 1998-01-13 Dainippon Printing Co Ltd 柱状基材への絵柄転写方法

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPS59130221A (ja) * 1982-10-26 1984-07-26 シ−テイエイ・フイナンツ・アクチエンゲゼルシヤフト ヒトと動物の遺伝学的変動域内における器官の組織物質の改良剤と改良方法
US7491857B2 (en) 2005-07-27 2009-02-17 National Institute Of Advanced Industrial Science And Technology Oligonucleotide probe

Also Published As

Publication number Publication date
EP0101985A1 (en) 1984-03-07
CA1202254A (en) 1986-03-25
US4667025A (en) 1987-05-19
DE3373817D1 (en) 1987-10-29
US4789737A (en) 1988-12-06
JPH0372639B2 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html) 1991-11-19
EP0101985B1 (en) 1987-09-23
US4820812A (en) 1989-04-11

Similar Documents

Publication Publication Date Title
JPS5927900A (ja) 固定化オリゴヌクレオチド
JP2533100B2 (ja) ポリヌクレオドの誘導方法
DE3854969T2 (de) Polynukleotid-Bestimmung mit selektierbaren Spaltstellen
US7572582B2 (en) Oligonucleotide analogues
US6794499B2 (en) Oligonucleotide analogues
US4948882A (en) Single-stranded labelled oligonucleotides, reactive monomers and methods of synthesis
JP3081882B2 (ja) 還元によるヒドロキシル保護基の直交除去及びオリゴヌクレオチドの化学合成におけるそれらの利用
US6410225B1 (en) Purification of oligomers
WO1996006104A1 (en) Polynucleotide reagents having nonnucleotidic moieties, and associated methods of synthesis and use
JPH05504564A (ja) アキラル内部サブユニット結合を有する非帯電モルホリノに基づくポリメラーゼ
EP1015469A2 (en) Bi- and tri-cyclic nucleoside, nucleotide and oligonucleoide analogues
JPH06211892A (ja) オリゴヌクレオチドの高分子支持体系
JPH0314319B2 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
US7098326B2 (en) Methods for the integrated synthesis and purification of oligonucleotides
JPH03504015A (ja) アルファ‐オリゴリボヌクレオチドの合成方法およびこの方法に用いられる化合物
JPH0435480B2 (GUID-C5D7CC26-194C-43D0-91A1-9AE8C70A9BFF.html)
JPS5993100A (ja) オリゴヌクレオチド誘導体
JPS6133195A (ja) オリゴヌクレオチド誘導体およびその製造法
JPS60166694A (ja) オリゴヌクレオチド誘導体およびその製造法
JPS60166695A (ja) オリゴヌクレオチド誘導体およびその製造法
OIJIGODEOXYRIBONUCI IJ. De Napoli, Al {essere, Dl, tontesarchio, G. Piccialli and
JPH05507619A (ja) Ｒｎａ加水分解／開裂