DE69404289T2 - Acyclische nucleosid analoge und sie enthaltende oligonucleotidsequenzen - Google Patents

Acyclische nucleosid analoge und sie enthaltende oligonucleotidsequenzen

Info

Publication number
DE69404289T2
DE69404289T2 DE69404289T DE69404289T DE69404289T2 DE 69404289 T2 DE69404289 T2 DE 69404289T2 DE 69404289 T DE69404289 T DE 69404289T DE 69404289 T DE69404289 T DE 69404289T DE 69404289 T2 DE69404289 T2 DE 69404289T2
Authority
DE
Germany
Prior art keywords
hydrogen
alkyl
image
amino
nucleoside analogs
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
DE69404289T
Other languages
English (en)
Other versions
DE69404289D1 (de
Inventor
Philip Cook
Daniel Delecki
Charles Guinosso
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.)
Sanofi SA
Original Assignee
Sanofi SA
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 Sanofi SA filed Critical Sanofi SA
Application granted granted Critical
Publication of DE69404289D1 publication Critical patent/DE69404289D1/de
Publication of DE69404289T2 publication Critical patent/DE69404289T2/de
Anticipated expiration legal-status Critical
Expired - Fee Related legal-status Critical Current

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D473/00—Heterocyclic compounds containing purine ring systems
    • C07D473/02—Heterocyclic compounds containing purine ring systems with oxygen, sulphur, or nitrogen atoms directly attached in positions 2 and 6
    • C07D473/18—Heterocyclic compounds containing purine ring systems with oxygen, sulphur, or nitrogen atoms directly attached in positions 2 and 6 one oxygen and one nitrogen atom, e.g. guanine
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D473/00—Heterocyclic compounds containing purine ring systems
    • C07D473/26—Heterocyclic compounds containing purine ring systems with an oxygen, sulphur, or nitrogen atom directly attached in position 2 or 6, but not in both
    • C07D473/32—Nitrogen atom
    • C07D473/34—Nitrogen atom attached in position 6, e.g. adenine
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07F—ACYCLIC, CARBOCYCLIC OR HETEROCYCLIC COMPOUNDS CONTAINING ELEMENTS OTHER THAN CARBON, HYDROGEN, HALOGEN, OXYGEN, NITROGEN, SULFUR, SELENIUM OR TELLURIUM
    • C07F9/00—Compounds containing elements of Groups 5 or 15 of the Periodic System
    • C07F9/02—Phosphorus compounds
    • C07F9/547—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom
    • C07F9/6561—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings
    • C07F9/65616—Heterocyclic compounds, e.g. containing phosphorus as a ring hetero atom containing systems of two or more relevant hetero rings condensed among themselves or condensed with a common carbocyclic ring or ring system, with or without other non-condensed hetero rings containing the ring system having three or more than three double bonds between ring members or between ring members and non-ring members, e.g. purine or analogs
    • 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
DE69404289T 1993-03-30 1994-03-21 Acyclische nucleosid analoge und sie enthaltende oligonucleotidsequenzen Expired - Fee Related DE69404289T2 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US4032693A 1993-03-30 1993-03-30
PCT/US1994/002995 WO1994022864A1 (en) 1993-03-30 1994-03-21 Acyclic nucleoside analogs and oligonucleotide sequences containing them

Publications (2)

Publication Number Publication Date
DE69404289D1 DE69404289D1 (de) 1997-08-21
DE69404289T2 true DE69404289T2 (de) 1998-02-19

Family

ID=21910387

Family Applications (1)

Application Number Title Priority Date Filing Date
DE69404289T Expired - Fee Related DE69404289T2 (de) 1993-03-30 1994-03-21 Acyclische nucleosid analoge und sie enthaltende oligonucleotidsequenzen

Country Status (11)

Country Link
US (1) US5576427A (de)
EP (1) EP0691968B1 (de)
JP (1) JPH08508492A (de)
AT (1) ATE155467T1 (de)
AU (1) AU6449394A (de)
CA (1) CA2159631A1 (de)
DE (1) DE69404289T2 (de)
DK (1) DK0691968T3 (de)
ES (1) ES2107205T3 (de)
GR (1) GR3025053T3 (de)
WO (1) WO1994022864A1 (de)

Families Citing this family (645)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
EP0728139B1 (de) 1993-09-03 2003-08-13 Isis Pharmaceuticals, Inc. Aminoderivatisierte nukleoside und oligonukleoside
DE59607750D1 (de) * 1995-03-13 2001-10-31 Aventis Pharma Gmbh Phosphonomonoesternnukleinsäuren, Verfahren zu ihrer Herstellung und ihre Verwendung
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
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
US20040161777A1 (en) * 1996-06-06 2004-08-19 Baker Brenda F. Modified oligonucleotides for use in RNA interference
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
US20050118605A9 (en) * 1996-06-06 2005-06-02 Baker Brenda F. Oligomeric compounds having modified bases for binding to adenine and guanine and their use in gene modulation
US20040171030A1 (en) * 1996-06-06 2004-09-02 Baker Brenda F. Oligomeric compounds having modified bases for binding to cytosine and uracil or thymine and their use in gene modulation
US20040171032A1 (en) * 1996-06-06 2004-09-02 Baker Brenda F. Non-phosphorous-linked oligomeric compounds and their use in gene modulation
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
AU6452098A (en) 1997-03-07 1998-09-22 Metabasis Therapeutics, Inc. Novel purine inhibitors of fructose-1,6-bisphosphatase
DE69827060T2 (de) * 1997-03-20 2005-03-24 F. Hoffmann-La Roche Ag Modifizierte Primer
DE69834038D1 (de) 1997-07-01 2006-05-18 Isis Pharmaceutical Inc Zusammensetzungen und verfahren zur verabreichung von oligonukleotiden über die speiseröhre
US20040186071A1 (en) 1998-04-13 2004-09-23 Bennett C. Frank Antisense modulation of CD40 expression
EP1469009A2 (de) * 1998-05-21 2004-10-20 Isis Parmaceuticals, Inc. Zusammensetzungen und Verfahren für nicht-parenterale Verabreichung von Oligonukleotiden
EP1080226A4 (de) 1998-05-21 2004-04-21 Isis Pharmaceuticals Inc Zusammensetzungen und methoden zur topischen verabreichung von oligonukleotiden
US6335439B1 (en) 1998-06-11 2002-01-01 Isis Pharmaceuticals, Inc. Method of preparing phosphoramidites
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
US6300320B1 (en) 1999-01-05 2001-10-09 Isis Pharmaceuticals, Inc. Modulation of c-jun using inhibitors of protein kinase C
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
US6147200A (en) * 1999-08-19 2000-11-14 Isis Pharmaceuticals, Inc. 2'-O-acetamido modified monomers and oligomers
US6617442B1 (en) 1999-09-30 2003-09-09 Isis Pharmaceuticals, Inc. Human Rnase H1 and oligonucleotide compositions thereof
US7668658B2 (en) 1999-10-13 2010-02-23 Sequenom, Inc. Methods for generating databases and databases for identifying polymorphic genetic markers
AU1916201A (en) * 1999-11-08 2001-06-06 Origenix Technologies, Inc. Combinatorial library synthesis and pharmaceutically active compounds produced thereby
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
US20020127598A1 (en) * 2000-03-27 2002-09-12 Wenqiang Zhou Solution-phase combinatorial library synthesis and pharmaceutically active compounds produced thereby
JP2003530841A (ja) * 2000-04-13 2003-10-21 トーマス エヌ． ワイト， 治療的化合物および方法
US6680172B1 (en) 2000-05-16 2004-01-20 Regents Of The University Of Michigan Treatments and markers for cancers of the central nervous system
US6958214B2 (en) 2000-07-10 2005-10-25 Sequenom, Inc. Polymorphic kinase anchor proteins and nucleic acids encoding the same
WO2002008446A2 (en) * 2000-07-13 2002-01-31 Origenix Technologies, Inc. Synthesis and antiviral evaluation of nucleic acid based (nab) libraries
US8568766B2 (en) 2000-08-24 2013-10-29 Gattadahalli M. Anantharamaiah Peptides and peptide mimetics to treat pathologies associated with eye disease
US6548251B1 (en) 2000-09-05 2003-04-15 Fidelity Systems, Inc. Inhibition of molecular and biological processes using modified oligonucleotides
AU2001296846B2 (en) 2000-10-12 2007-07-05 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
ATE434936T1 (de) 2001-03-14 2009-07-15 Myriad Genetics Inc Tsg101-gag-wechselwirkung und ihre verwendung
JP2005525991A (ja) 2001-05-16 2005-09-02 マイジェニックス インコーポレイテッド 核酸ベースの化合物およびその使用方法
US7803915B2 (en) 2001-06-20 2010-09-28 Genentech, Inc. Antibody compositions for the diagnosis and treatment of tumor
EP2000148A1 (de) 2001-06-20 2008-12-10 Genentech, Inc. Zusammensetzungen und Verfahren zur Behandlung und Diagnose von Prostatakarzinom
EP1404698A4 (de) 2001-06-21 2004-12-22 Isis Pharmaceuticals Inc Antisense-modulation von superoxiddismutase 1, expression in lösung
US6964950B2 (en) 2001-07-25 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of C-reactive protein expression
US7425545B2 (en) 2001-07-25 2008-09-16 Isis Pharmaceuticals, Inc. 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
NZ531674A (en) 2001-09-18 2009-03-31 Genentech Inc Compositions and methods for the diagnosis and treatment of tumor
US6750019B2 (en) 2001-10-09 2004-06-15 Isis Pharmaceuticals, Inc. Antisense modulation of insulin-like growth factor binding protein 5 expression
EP2270231A3 (de) 2001-10-09 2011-04-27 ISIS Pharmaceuticals, Inc. Antisense Modulation der Expression von insulin-like growth factor binding protein 5
US6965025B2 (en) 2001-12-10 2005-11-15 Isis Pharmaceuticals, Inc. Antisense modulation of connective tissue growth factor expression
EP2067472A1 (de) 2002-01-02 2009-06-10 Genentech, Inc. Zusammensetzungen und Verfahren zur Behandlung und Diagnose von Tumoren
US7553619B2 (en) 2002-02-08 2009-06-30 Qiagen Gmbh Detection method using dissociated rolling circle amplification
US7169916B2 (en) * 2002-04-01 2007-01-30 Isis Pharmaceuticals, Inc. Chloral-free DCA in oligonucleotide synthesis
NZ535925A (en) 2002-04-16 2008-06-30 Genentech Inc An isolated antibody that binds to a particular polypeptide
US7432342B2 (en) 2002-05-03 2008-10-07 Sequenom, Inc. Kinase anchor protein muteins, peptides thereof and related documents
US7199107B2 (en) 2002-05-23 2007-04-03 Isis Pharmaceuticals, Inc. Antisense modulation of kinesin-like 1 expression
CA2495478A1 (en) 2002-08-05 2004-02-12 University Of Rochester Protein transducing domain/deaminase chimeric proteins, related compounds, and uses thereof
EP2330194A3 (de) 2002-09-13 2011-10-12 Replicor, Inc. Nicht-sequenzkomplementäre antivirale Oligonukleotide
CA2499770A1 (en) 2002-09-20 2004-04-01 Yale University Riboswitches, methods for their use, and compositions for use with riboswitches.
EP1549767A4 (de) 2002-09-26 2006-06-07 Amgen Inc Modulation der expression von forkhead-box o1a
US9150606B2 (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
AU2003291753B2 (en) * 2002-11-05 2010-07-08 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
WO2004042029A2 (en) * 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Oligomers comprising modified bases for binding cytosine and uracil or thymine and their use
US9150605B2 (en) * 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
EP2336318B1 (de) 2002-11-13 2013-04-24 Genzyme Corporation Antisense-modulation von apolipoprotein-b-expression
DK1569695T3 (da) 2002-11-13 2013-08-05 Genzyme Corp Antisense-modulering af apolipoprotein-b-ekspression
EP1572971B1 (de) 2002-11-15 2009-09-30 Morphotek Inc. Verfahren zur erzeugung einer hohen antikörperproduktion von durch in-vitro-immunisierung entstandenen hybridomen
CN100594037C (zh) 2002-11-15 2010-03-17 Musc研究发展基金会 通过补体受体2定向的补体调节剂
EP1624753B1 (de) 2002-11-21 2012-01-25 The University of Utah Research Foundation Purinerge geruchsmodulation
US7144999B2 (en) 2002-11-23 2006-12-05 Isis Pharmaceuticals, Inc. Modulation of hypoxia-inducible factor 1 alpha expression
US9487823B2 (en) 2002-12-20 2016-11-08 Qiagen Gmbh Nucleic acid amplification
JP4886298B2 (ja) 2002-12-20 2012-02-29 キアゲン ゲゼルシャフト ミット ベシュレンクテル ハフツング 核酸増幅
US6977153B2 (en) * 2002-12-31 2005-12-20 Qiagen Gmbh Rolling circle amplification of RNA
DK1597366T3 (da) 2003-02-11 2013-02-25 Antisense Therapeutics Ltd Modulering af ekspression af insulin-lignende vækstfaktor receptor I
US7002006B2 (en) * 2003-02-12 2006-02-21 Isis Pharmaceuticals, Inc. Protection of nucleosides
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
WO2005002507A2 (en) 2003-06-03 2005-01-13 Isis Pharmaceuticals, Inc. Modulation of survivin expression
GB2417727B (en) 2003-06-13 2008-01-16 Alnylam Europe Ag Double-stranded ribonucleic acid with increased effectiveness in an organism
EP1644475A4 (de) * 2003-06-20 2009-06-03 Isis Pharmaceuticals Inc Doppelstrang-zusammensetzungen mit einem 3'-endo-modifizierten strang zur verwendung bei der genmodulation
CA2533701A1 (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
CA2538252C (en) 2003-09-18 2014-02-25 Isis Pharmaceuticals, Inc. 4'-thionucleosides and oligomeric compounds
WO2005028628A2 (en) 2003-09-18 2005-03-31 Isis Pharmaceuticals, Inc. Modulation of eif4e expression
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
JP2007520222A (ja) 2004-01-20 2007-07-26 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 グルココルチコイドレセプター発現の調節
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
EP2700720A3 (de) 2004-03-15 2015-01-28 Isis Pharmaceuticals, Inc. Zusammensetzungen und Verfahren zur Optimierung der Spaltung von RNA durch RNase H
US20050244869A1 (en) 2004-04-05 2005-11-03 Brown-Driver Vickie L Modulation of transthyretin expression
CA2561741C (en) 2004-04-05 2016-09-27 Alnylam Pharmaceuticals, Inc. Processes and reagents for oligonucleotide synthesis and purification
EP1750776A2 (de) 2004-04-30 2007-02-14 Alnylam Pharmaceuticals Inc. Oligonukleotide mit c5-modifiziertem pyrimidin
EP3225633B1 (de) 2004-05-21 2020-03-25 The UAB Research Foundation Variable lymphozytenrezeptoren, verwandte polypeptide und nukleinsäuren sowie verwendung dafür
US8394947B2 (en) 2004-06-03 2013-03-12 Isis Pharmaceuticals, Inc. Positionally modified siRNA constructs
US7427675B2 (en) 2004-08-23 2008-09-23 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
TR201907874T4 (tr) 2004-09-23 2019-06-21 Arc Medical Devices Inc Düşük sülfat fukanlar kullanarak fibröz yapışmaları ya da inflamatuar hastalıkları inhibe etmeye yönelik farmasötik kompozisyonlar ve yöntemler.
ES2392449T3 (es) 2004-10-20 2012-12-10 Antisense Therapeutics Ltd Modulación antisentido de la expresión de la integrina alfa-4
US20100256062A1 (en) * 2004-12-06 2010-10-07 Howard Tommy E Allelic Variants of Human Factor VIII
CN101175769A (zh) 2005-03-10 2008-05-07 健泰科生物技术公司 用于调控血管完整性的方法和组合物
EP1863908B1 (de) 2005-04-01 2010-11-17 Qiagen GmbH Reverse transkription und amplifikation von rna bei simultaner degradierung von dna
JP5329949B2 (ja) 2005-05-31 2013-10-30 エコーレ ポリテクニーク フェデラーレ デ ローザンヌ 遺伝子に基づいた薬物の細胞質送達のためのトリブロックコポリマー
WO2006133022A2 (en) 2005-06-03 2006-12-14 The Johns Hopkins University Compositions and methods for decreasing microrna expression for the treatment of neoplasia
US8252756B2 (en) 2005-06-14 2012-08-28 Northwestern University Nucleic acid functionalized nanoparticles for therapeutic applications
ATE483802T1 (de) 2005-08-11 2010-10-15 Synthetic Genomics Inc Verfahren zur in vitro-rekombination
JP2009504168A (ja) 2005-08-17 2009-02-05 メディクシス エス．エー． Ｃｋ１９発現を確認する組成物及び方法
EP2338992A3 (de) 2005-08-29 2011-10-12 Regulus Therapeutics, Inc Antisense-verbindungen mit verbesserter anti-mikrorna-aktivität
WO2007027775A2 (en) 2005-08-29 2007-03-08 Isis Pharmaceuticals, Inc. Methods for use in modulating mir-122a
EP1762627A1 (de) 2005-09-09 2007-03-14 Qiagen GmbH Verfahren zur Aktivierung einer Nukleinsäure für eine Polymerase-Reaktion
AU2006304321B2 (en) 2005-10-14 2012-10-04 Musc Foundation For Research Development Targeting PAX2 for the induction of DEFB1-mediated tumor immunity and cancer therapy
US8080534B2 (en) 2005-10-14 2011-12-20 Phigenix, Inc Targeting PAX2 for the treatment of breast cancer
WO2007051045A2 (en) * 2005-10-28 2007-05-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of huntingtin gene
AU2006311730B2 (en) 2005-11-09 2010-12-02 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
EP1974052A2 (de) 2005-12-21 2008-10-01 Yale University Verfahren und zusammensetzungen in verbindung mit der modulation von riboschaltern
US8288354B2 (en) 2005-12-28 2012-10-16 The Scripps Research Institute Natural antisense and non-coding RNA transcripts as drug targets
WO2007089584A2 (en) 2006-01-26 2007-08-09 Isis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
EP2388327A1 (de) 2006-01-27 2011-11-23 Isis Pharmaceuticals, Inc. Oligomerverbindungen und Zusammensetzungen zur Verwendung bei der Modulation von microRNAs
CA2640171C (en) 2006-01-27 2014-10-28 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
US7569686B1 (en) 2006-01-27 2009-08-04 Isis Pharmaceuticals, Inc. Compounds and methods for synthesis of bicyclic nucleic acid analogs
KR20150038522A (ko) 2006-03-31 2015-04-08 알닐람 파마슈티칼스 인코포레이티드 Ｅｇ5 유전자의 발현을 억제하는 이본쇄 리보핵산
JP2009535383A (ja) 2006-05-03 2009-10-01 バルティック テクロノジー デヴェロプメント，リミテッド 強く結合した塩基で修飾されたオリゴヌクレオチドと人工ヌクレアーゼを組み合わせたアンチセンス作用物質
AU2007253909B2 (en) 2006-05-05 2012-08-09 Isis Pharmaceuticals, Inc. Compounds and methods for modulating expression of GCCR
EP2505647A1 (de) 2006-05-05 2012-10-03 Isis Pharmaceuticals, Inc. Verbindungen und Verfahren zur Modulation der Expression von DGAT2
US7666854B2 (en) 2006-05-11 2010-02-23 Isis Pharmaceuticals, Inc. Bis-modified bicyclic nucleic acid analogs
EP2066684B1 (de) 2006-05-11 2012-07-18 Isis Pharmaceuticals, Inc. 5'-modifizierte bicyclische nukleinsäureanaloga
JP2009537153A (ja) 2006-05-19 2009-10-29 アルニラム ファーマシューティカルズ， インコーポレイテッド ＡｈａのＲＮＡｉ調節およびその治療上の使用
US7888498B2 (en) 2006-05-22 2011-02-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of IKK-B gene
US8198253B2 (en) 2006-07-19 2012-06-12 Isis Pharmaceuticals, Inc. Compositions and their uses directed to HBXIP
JP2010500290A (ja) * 2006-08-04 2010-01-07 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 Ｊｎｋタンパク質を調節するための組成物および方法
US7947487B2 (en) 2006-10-05 2011-05-24 Massachusetts Institute Of Technology Multifunctional encoded particles for high-throughput analysis
US9550988B2 (en) 2006-10-18 2017-01-24 Ionis Pharmaceuticals, Inc. Antisense compounds
WO2008136852A2 (en) 2006-11-01 2008-11-13 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
JP2010510807A (ja) 2006-11-27 2010-04-08 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 高コレステロール血症を治療するための方法
US7994130B2 (en) 2006-12-11 2011-08-09 University Of Utah Research Foundation Compositions and methods for treating ocular pathologic angiogenesis and vascular permeability
EP2097448A4 (de) 2006-12-22 2010-07-21 Univ Utah Res Found Verfahren für den nachweis von augenerkrankungen und leiden sowie ihre behandlung
MX2009008470A (es) 2007-02-09 2009-11-26 Univ Northwestern Particulas para detectar objetivos intracelulares.
CN101801185A (zh) 2007-03-22 2010-08-11 耶鲁大学 与控制可变剪接的核糖开关有关的方法和组合物
AU2008232891B2 (en) 2007-03-29 2012-01-12 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a gene from the Ebola
US20110091377A1 (en) 2007-05-11 2011-04-21 The Johns Hopkins University Biomarkers for melanoma
US20100286082A1 (en) 2007-05-29 2010-11-11 Yale University Riboswitches and methods and compositions for use of and with riboswitches
CA2688321A1 (en) 2007-05-30 2008-12-11 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
AU2008259907B2 (en) 2007-05-30 2014-12-04 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
ES2386492T3 (es) 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
AU2008272918B2 (en) 2007-07-05 2012-09-13 Isis Pharmaceuticals, Inc. 6-disubstituted bicyclic nucleic acid analogs
US8088904B2 (en) 2007-08-15 2012-01-03 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
AU2008296487A1 (en) 2007-08-28 2009-03-12 The Uab Research Foundation Synthetic apolipoprotein E mimicking polypeptides and methods of use
AU2008296478B9 (en) 2007-08-28 2015-03-19 The 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
ES2641290T3 (es) 2007-11-20 2017-11-08 Ionis Pharmaceuticals, Inc Modulación de la expresión de CD40
EP2848688A1 (de) 2007-12-10 2015-03-18 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und Verfahren zur Hemmung der Faktor VII-Genexpression
US20110117125A1 (en) 2008-01-02 2011-05-19 Tekmira Pharmaceuticals Corporation Compositions and methods for the delivery of nucleic acids
US8530640B2 (en) 2008-02-07 2013-09-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
MX2010009611A (es) 2008-03-05 2010-12-15 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de genes eg5 y vegf.
WO2009117589A1 (en) 2008-03-21 2009-09-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising tricyclic nucleosides and methods for their use
WO2009124238A1 (en) 2008-04-04 2009-10-08 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising neutrally linked terminal bicyclic nucleosides
US8846639B2 (en) 2008-04-04 2014-09-30 Isis Pharmaceutical, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
DK2281042T3 (en) 2008-04-18 2015-11-09 Baxter Int Microsphere-based composition for preventing and / or deletion of starting autoimmune diabetes
US20090274696A1 (en) * 2008-04-29 2009-11-05 Wyeth Methods for treating inflammation
US8222221B2 (en) 2008-06-04 2012-07-17 The Board Of Regents Of The University Of Texas System Modulation of gene expression through endogenous small RNA targeting of gene promoters
JP2011529686A (ja) 2008-07-29 2011-12-15 ザ ボード オブ リージェンツ オブ ザ ユニバーシティー オブ テキサス システム ポリグルタミンタンパク質発現の選択的阻害
ES2657696T3 (es) 2008-08-25 2018-03-06 Excaliard Pharmaceuticals, Inc. Método para reducir la cicatrización durante la curación de una herida utilizando compuestos antisentido dirigidos al CTGF
KR101877698B1 (ko) 2008-08-25 2018-07-12 엑스칼리아드 파마슈티컬즈, 인코포레이티드 결합 조직 성장 인자에 대한 안티센스 올리고뉴클레오타이드 및 그의 용도
US8318693B2 (en) 2008-09-02 2012-11-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of mutant EGFR gene
EP2356129B1 (de) 2008-09-24 2013-04-03 Isis Pharmaceuticals, Inc. Substituierte alpha-l-bicyclische nukleoside
DK2361256T3 (da) 2008-09-24 2013-07-01 Isis Pharmaceuticals Inc Cyclohexenyl-nukleinsyreanaloger
ES2740129T3 (es) 2008-09-25 2020-02-05 Alnylam Pharmaceuticals Inc Composiciones formuladas en lípidos y métodos de inhibición de la expresión del gen de suero amiloide A
EP3225621A1 (de) 2008-10-09 2017-10-04 Arbutus Biopharma Corporation Verbesserte aminolipide und verfahren zur freisetzung von nukleinsäuren
CN104212799B (zh) 2008-10-15 2018-11-23 Ionis制药公司 因子11表达的调节
KR102354558B1 (ko) 2008-10-20 2022-01-25 알닐람 파마슈티칼스 인코포레이티드 트랜스티레틴의 발현을 억제하기 위한 조성물 및 방법
US20120059045A1 (en) 2008-10-24 2012-03-08 Isis Pharmaceuticals, Inc. Methods of using oligomeric compounds comprising 2'-substituted nucleosides
US8987435B2 (en) 2008-10-24 2015-03-24 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
CN106955360A (zh) 2008-11-24 2017-07-18 西北大学 多价rna纳米颗粒组合物
WO2010065662A2 (en) 2008-12-04 2010-06-10 Curna, Inc. Treatment of sirtuin 1 (sirt1) related diseases by inhibition of natural antisense transcript to sirtuin 1
JP6091752B2 (ja) 2008-12-04 2017-03-08 クルナ・インコーポレーテッド Ｅｐｏに対する天然アンチセンス転写物の抑制によるエリスロポエチン（ｅｐｏ）関連疾患の治療
ES2637063T3 (es) 2008-12-04 2017-10-10 Curna, Inc. Tratamiento de enfermedades relacionadas con genes supresores de tumor mediante inhibición del transcrito antisentido natural al gen
AU2009324534B2 (en) 2008-12-10 2015-07-30 Alnylam Pharmaceuticals, Inc. GNAQ targeted dsRNA compositions and methods for inhibiting expression
US20100233270A1 (en) 2009-01-08 2010-09-16 Northwestern University Delivery of Oligonucleotide-Functionalized Nanoparticles
AU2010208035B2 (en) 2009-01-29 2016-06-23 Arbutus Biopharma Corporation Improved lipid formulation for the delivery of nucleic acids
WO2010091308A2 (en) 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
US8536320B2 (en) 2009-02-06 2013-09-17 Isis Pharmaceuticals, Inc. Tetrahydropyran nucleic acid analogs
CN102387817B (zh) 2009-02-12 2018-01-30 库尔纳公司 通过抑制针对脑衍生神经营养因子(bdnf)的天然反义转录物来治疗bdnf相关的疾病
CN102439149B (zh) 2009-02-12 2018-01-02 库尔纳公司 通过抑制针对胶质细胞衍生神经营养因子(gdnf)的天然反义转录物来治疗gdnf相关的疾病
WO2010099341A1 (en) 2009-02-26 2010-09-02 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of mig-12 gene
ES2845644T3 (es) 2009-03-04 2021-07-27 Curna Inc Tratamiento de enfermedades relacionadas con sirtuina1 (SIRT1) mediante inhibición del transcrito del antisentido natural a sirtuina 1
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
CA2755409C (en) 2009-03-16 2019-04-30 Joseph Collard 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).
EP2419535B1 (de) 2009-04-15 2022-08-17 Northwestern University Freisetzung von oligonukleotid-funktionalisierten nanopartikeln
EP3524275A1 (de) 2009-04-22 2019-08-14 Massachusetts Institute Of Technology Angeborene immununterdrückung ermöglicht die wiederholte verabreichung von langen rna-molekülen
EP2421972A2 (de) 2009-04-24 2012-02-29 The Board of Regents of The University of Texas System Modulation der genexpression mit auf genregionen nach 3-untranslatierten regionen gerichteten oligomeren
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
CA2760706C (en) 2009-05-05 2019-08-06 Alnylam Pharmaceuticals, Inc. Methods of delivering oligonucleotides to immune cells
CN102421417B (zh) 2009-05-05 2016-03-02 阿尔尼拉姆医药品有限公司 脂质组合物
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
CN102803492B (zh) 2009-05-06 2016-06-29 库尔纳公司 通过抑制针对三重四脯氨酸(ttp)的天然反义转录物来治疗ttp相关疾病
WO2010132665A1 (en) 2009-05-15 2010-11-18 Yale University Gemm riboswitches, structure-based compound design with gemm riboswitches, and methods and compositions for use of and with gemm riboswitches
JP5922017B2 (ja) 2009-05-18 2016-05-24 クルナ・インコーポレーテッド リプログラミング因子に対する天然アンチセンス転写物の抑制によるリプログラミング因子関連疾患の治療
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
ES2618576T3 (es) 2009-05-28 2017-06-21 Curna, Inc. Tratamiento de enfermedades relacionadas con un gen antivírico mediante la inhibición de una transcripción antisentido natural a un gen antivírico
EP2440183B1 (de) 2009-06-10 2018-07-18 Arbutus Biopharma Corporation Verbesserte lipidformulierung
ES2629339T3 (es) 2009-06-16 2017-08-08 Curna, Inc. Tratamiento de enfermedades relacionadas con la paraoxonasa 1 (pon1) por inhibición de transcrito antisentido natural a pon1
KR101801404B1 (ko) 2009-06-16 2017-12-20 큐알엔에이, 인크. 콜라겐 유전자에 대한 천연 안티센스 전사체의 억제에 의한 콜라겐 유전자 관련된 질환의 치료
EP3305302B1 (de) 2009-06-17 2018-09-19 Biogen MA Inc. Zusammensetzungen und verfahren zur modulation der smn2-aufspaltung im körper eines patienten
CN102597238B (zh) 2009-06-24 2016-06-29 库尔纳公司 通过抑制针对肿瘤坏死因子受体2(tnfr2)的天然反义转录物来治疗tnfr2相关的疾病
WO2010151674A2 (en) 2009-06-26 2010-12-29 Curna, Inc. Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene
CA2769665A1 (en) 2009-08-05 2011-02-10 Opko Curna, Llc Treatment of insulin gene (ins) related diseases by inhibition of natural antisense transcript to an insulin gene (ins)
WO2011017521A2 (en) 2009-08-06 2011-02-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
AP3574A (en) 2009-08-14 2016-02-08 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
KR101892760B1 (ko) 2009-08-25 2018-08-28 큐알엔에이, 인크. ＩＱＧＡＰ에 대한 천연 안티센스 전사체의 억제에 의한 ＧＴＰａｓｅ 활성화 단백질을 함유하는 ＩＱ 모티프(ＩＱＧＡＰ)와 관련된 질환의 치료
WO2011028950A1 (en) 2009-09-02 2011-03-10 Genentech, Inc. Mutant smoothened and methods of using the same
KR20120105446A (ko) 2009-10-22 2012-09-25 제넨테크, 인크. 대식세포-자극 단백질의 헵신 활성화를 조정하기 위한 방법 및 조성물
CA2778532A1 (en) 2009-10-27 2011-05-12 Swift Biosciences, Inc. Polynucleotide primers and probes
JP5866119B2 (ja) 2009-10-30 2016-02-17 ノースウェスタン ユニバーシティ 鋳型ナノ複合体
WO2011053994A1 (en) 2009-11-02 2011-05-05 Alnylam Pharmaceuticals, Inc. Modulation of ldl receptor gene expression with double-stranded rnas targeting the ldl receptor gene promoter
KR20120102674A (ko) 2009-11-03 2012-09-18 유니버시티 오브 버지니아 페이턴트 파운데이션 중합체 분석물을 검출하는 다용도의 가시적인 방법
JP2013511285A (ja) 2009-11-23 2013-04-04 スイフト・バイオサイエンシズ・インコーポレイテツド 一本鎖標的分子を伸長させるデバイス
RU2015132142A (ru) 2009-11-30 2015-12-20 Дженентек, Инк. Композиции и способы для диагностики и лечения опухоли
ES2661813T3 (es) 2009-12-16 2018-04-04 Curna, Inc. Tratamiento de enfermedades relacionadas con la peptidasa del factor de transcripción de membrana, sitio 1 (mbtps1) mediante inhibición del transcrito antisentido natural al gen mbtps1
WO2011079263A2 (en) 2009-12-23 2011-06-30 Curna, Inc. Treatment of uncoupling protein 2 (ucp2) related diseases by inhibition of natural antisense transcript to ucp2
US8940708B2 (en) 2009-12-23 2015-01-27 Curna, Inc. Treatment of hepatocyte growth factor (HGF) related diseases by inhibition of natural antisense transcript to HGF
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
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
RU2611187C2 (ru) 2010-01-04 2017-02-21 Курна, Инк. Лечение заболеваний, связанных с интерферон-регуляторным фактором 8 (irf8), путем ингибирования природного антисмыслового транскрипта к irf8
RU2612161C2 (ru) 2010-01-06 2017-03-02 Курна, Инк. Лечение заболеваний, связанных с геном развития поджелудочной железы, путем ингибирования природного антисмыслового транскрипта к гену развития поджелудочной железы
RU2611191C2 (ru) 2010-01-11 2017-02-21 Курна, Инк. Лечение заболеваний, связанных со связывающим половые гормоны глобулином (гспг), путем ингибирования природного антисмыслового транскрипта к гспг
US8779118B2 (en) 2010-01-11 2014-07-15 Isis Pharmaceuticals, Inc. Base modified bicyclic nucleosides and oligomeric compounds prepared therefrom
WO2011088076A2 (en) 2010-01-12 2011-07-21 Yale University Structured rna motifs and compounds and methods for their use
US20110177107A1 (en) * 2010-01-14 2011-07-21 Haplomics, Inc. Predicting and reducing alloimmunogenicity of protein therapeutics
KR101853510B1 (ko) 2010-01-25 2018-06-20 큐알엔에이, 인크. Ｒｎａｓｅ ｈ1에 대한 천연 안티센스 전사체의 억제에 의한 ｒｎａｓｅ ｈ1과 관련된 질환의 치료
WO2011097388A1 (en) 2010-02-03 2011-08-11 Alnylam Pharmaceuticals, Inc. Selective inhibition of polyglutamine protein expression
US20130028889A1 (en) 2010-02-04 2013-01-31 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
WO2011105901A2 (en) 2010-02-23 2011-09-01 Academisch Ziekenhuis Bij De Universiteit Van Amsterdam Antagonists of complement component 9 (c9) and uses thereof
CA2787657A1 (en) 2010-02-23 2011-09-01 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
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
WO2011112516A1 (en) 2010-03-08 2011-09-15 Ico Therapeutics Inc. Treating and preventing hepatitis c virus infection using c-raf kinase antisense oligonucleotides
WO2011113054A2 (en) 2010-03-12 2011-09-15 Aurasense Llc Crosslinked polynucleotide structure
WO2011115818A1 (en) 2010-03-17 2011-09-22 Isis Pharmaceuticals, Inc. 5'-substituted bicyclic nucleosides and oligomeric compounds prepared therefrom
JP5860029B2 (ja) 2010-03-29 2016-02-16 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． トランスチレチン（ＴＴＲ）関連眼アミロイドーシスのためのｓｉＲＮＡ療法
CN102858979B (zh) 2010-04-09 2018-01-26 库尔纳公司 通过抑制成纤维细胞生长因子21(fgf21)的天然反义转录物而治疗fgf21相关疾病
US20110269194A1 (en) 2010-04-20 2011-11-03 Swift Biosciences, Inc. Materials and methods for nucleic acid fractionation by solid phase entrapment and enzyme-mediated detachment
US10913767B2 (en) 2010-04-22 2021-02-09 Alnylam Pharmaceuticals, Inc. Oligonucleotides comprising acyclic and abasic nucleosides and analogs
KR101869570B1 (ko) 2010-04-28 2018-06-20 아이오니스 파마수티컬즈, 인코포레이티드 변형된 뉴클레오사이드 및 그로부터 제조된 올리고머 화합물
EP3091027B1 (de) 2010-04-28 2018-01-17 Ionis Pharmaceuticals, Inc. 5'-modifizierte nukleoside und daraus hergestellte oligomere verbindungen
MX343559B (es) 2010-04-29 2016-11-10 Ionis Pharmaceuticals Inc Modulacion de la expresion de transtiretina.
KR20130101442A (ko) 2010-05-03 2013-09-13 큐알엔에이, 인크. 시르투인 (sirt)에 대한 자연 안티센스 전사체의 저해에 의한 시르투인 (sirt) 관련된 질환의 치료
CA2793544A1 (en) 2010-05-03 2011-11-10 Genentech, Inc. Compositions and methods for the diagnosis and treatment of tumor
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
KR20180053419A (ko) 2010-05-26 2018-05-21 큐알엔에이, 인크. 무조(無調)의 동소체 1 (atoh1)에 대한 자연 안티센스 전사체의 저해에 의한 atoh1 관련된 질환의 치료
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
WO2011156434A2 (en) 2010-06-07 2011-12-15 Firefly Bioworks, Inc. Nucleic acid detection and quantification by post-hybridization labeling and universal encoding
EP2580228B1 (de) 2010-06-08 2016-03-23 Ionis Pharmaceuticals, Inc. Substituierte 2 '-amino- und 2 '-thio-bicyclische nukleoside und daraus hergestellte oligomere verbindungen
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)
DK2593547T3 (en) 2010-07-14 2018-02-26 Curna Inc Treatment of Discs large homolog (DLG) related diseases by inhibition of natural antisense transcript to DLG
EP2595664B1 (de) 2010-07-19 2018-10-17 Ionis Pharmaceuticals, Inc. Modulation einer kern-rna
WO2012021554A1 (en) 2010-08-09 2012-02-16 Yale University Cyclic di-gmp-ii riboswitches, motifs, and compounds, and methods for their use
AU2011312205B2 (en) 2010-10-05 2015-08-13 Curis, Inc. Mutant smoothened and methods of using the same
ES2640755T3 (es) 2010-10-06 2017-11-06 Curna, Inc. Tratamiento de enfermedades relacionadas con la Sialidasa 4 (neu4) mediante inhibición del transcrito antisentido natural al gen neu4
EP2630241B1 (de) 2010-10-22 2018-10-17 CuRNA, Inc. Behandlung von alpha-l-iduronidase (idua)-bedingten erkrankungen mittels hemmung des natürlichen antisense-transkripts gegen idua
CA2816056A1 (en) 2010-10-27 2012-05-03 Curna, Inc. Treatment of interferon-related developmental regulator 1 (ifrd1) related diseases by inhibition of natural antisense transcript to ifrd1
WO2012064824A1 (en) 2010-11-09 2012-05-18 Alnylam Pharmaceuticals, Inc. Lipid formulated compositions and methods for inhibiting expression of eg5 and vegf genes
CA2817960C (en) 2010-11-17 2020-06-09 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
US8987225B2 (en) 2010-11-23 2015-03-24 Curna, Inc. Treatment of NANOG related diseases by inhibition of natural antisense transcript to NANOG
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
US9193973B2 (en) 2010-12-10 2015-11-24 Alynylam Pharmaceuticals, Inc. Compositions and methods for increasing erythropoietin (EPO) production
EP2648763A4 (de) 2010-12-10 2014-05-14 Alnylam Pharmaceuticals Inc Zusammensetzungen und verfahren zur expressionshemmung der gene klf-1 und bcl11a
AU2012212110A1 (en) 2011-02-02 2013-08-01 Excaliard Pharmaceuticals, Inc. Method of treating keloids or hypertrophic scars using antisense compounds targeting connective tissue growth factor (CTGF)
CN103391777A (zh) 2011-02-02 2013-11-13 普林斯顿大学理事会 作为病毒产生调节剂的去乙酰化酶调节剂
KR20140020900A (ko) 2011-02-03 2014-02-19 미르나 테라퓨틱스 인코포레이티드 Ｍｉｒ―34의 합성 모방체
US9222085B2 (en) 2011-02-03 2015-12-29 Mirna Therapeutics, Inc. Synthetic mimics of MIR-124
EP2673361B1 (de) 2011-02-08 2016-04-13 Ionis Pharmaceuticals, Inc. Oligomere verbindungen mit bicyclischen nukleotiden und ihre verwendung
US9562853B2 (en) 2011-02-22 2017-02-07 Vanderbilt University Nonaqueous backscattering interferometric methods
KR102104401B1 (ko) 2011-03-29 2020-04-27 알닐람 파마슈티칼스 인코포레이티드 Tmprss6 유전자의 발현을 억제하기 위한 조성물 및 방법
US10086043B2 (en) 2011-04-03 2018-10-02 The General Hospital Corporation Efficient protein expression in vivo using modified RNA (MOD-RNA)
WO2012149154A1 (en) 2011-04-26 2012-11-01 Swift Biosciences, Inc. Polynucleotide primers and probes
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
WO2012151289A2 (en) 2011-05-02 2012-11-08 University Of Virginia Patent Foundation Method and system to detect aggregate formation on a substrate
WO2012170771A1 (en) 2011-06-09 2012-12-13 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
WO2012170945A2 (en) 2011-06-10 2012-12-13 Isis Pharmaceuticals, Inc. Methods for modulating kallikrein (klkb1) expression
BR122019026068B8 (pt) 2011-06-21 2022-10-18 Alnylam Pharmaceuticals Ácido ribonucleico de fita dupla (dsrna) para inibir a expressão de angptl3 e seu uso, composição farmacêutica e método in vitro para inibir a expressão de angptl3 em uma célula
KR102540778B1 (ko) 2011-06-21 2023-06-07 알닐람 파마슈티칼스 인코포레이티드 아포리포단백질 c-iii(apoc3) 유전자의 발현 억제를 위한 조성물 및 방법
WO2012177949A2 (en) 2011-06-21 2012-12-27 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibition of expression of protein c (proc) genes
EP4134433A1 (de) 2011-06-23 2023-02-15 Alnylam Pharmaceuticals, Inc. Serpina1-sirnas: materialzusammensetzungen und behandlungsverfahren
US9322021B2 (en) 2011-06-29 2016-04-26 Ionis Pharmaceuticals, Inc. Methods for modulating kallikrein (KLKB1) expression
WO2013019857A2 (en) 2011-08-01 2013-02-07 Alnylam Pharmaceuticals, Inc. Method for improving the success rate of hematopoietic stem cell transplants
WO2013022967A1 (en) 2011-08-11 2013-02-14 Isis Pharmaceuticals, Inc. Gapped oligomeric compounds comprising 5'-modified deoxyribonucleosides in the gap and uses thereof
WO2013040499A1 (en) 2011-09-14 2013-03-21 Northwestern University Nanoconjugates able to cross the blood-brain barrier
WO2013040548A2 (en) 2011-09-17 2013-03-21 Yale University Fluoride-responsive riboswitchs, fluoride transporters, and methods of use
ES2687951T3 (es) 2011-10-14 2018-10-30 F. Hoffmann-La Roche Ag Anticuerpos anti-HtrA1 y procedimientos de uso
WO2013090457A2 (en) 2011-12-12 2013-06-20 Oncoimmunin Inc. In vivo delivery of oligonucleotides
CA3018046A1 (en) 2011-12-16 2013-06-20 Moderna Therapeutics, Inc. Modified nucleoside, nucleotide, and nucleic acid compositions
JP6280045B2 (ja) 2011-12-22 2018-02-14 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． 肺腺癌内転移関連性転写物１（ｍｅｔａｓｔａｓｉｓ−ａｓｓｏｃｉａｔｅｄ−ｉｎ−ｌｕｎｇ−ａｄｅｎｏｃａｒｃｉｎｏｍａ−ｔｒａｎｓｃｒｉｐｔ−１：ｍａｌａｔ−１）の発現調節法
WO2013106358A1 (en) 2012-01-10 2013-07-18 Hussain M Mahmood Method of treating hyperlipidemia and atherosclerosis with mir-30c
EP2812342B1 (de) 2012-02-08 2017-11-15 Ionis Pharmaceuticals, Inc. Modulation von rna durch wiederholtes targeting
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
CN110438125A (zh) 2012-03-15 2019-11-12 科纳公司 通过抑制脑源神经营养因子(bdnf)的天然反义转录物治疗bdnf相关疾病
CA2907072A1 (en) 2012-03-16 2013-09-19 Valerion Therapeutics, Llc Antisense conjugates for decreasing expression of dmpk
US9340784B2 (en) 2012-03-19 2016-05-17 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating alpha-1-antitrypsin expression
AU2013243950A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides
AU2013243949A1 (en) 2012-04-02 2014-10-30 Moderna Therapeutics, Inc. Modified polynucleotides for the production of biologics and proteins associated with human disease
US9221864B2 (en) 2012-04-09 2015-12-29 Isis Pharmaceuticals, Inc. Tricyclic nucleic acid analogs
WO2013154799A1 (en) 2012-04-09 2013-10-17 Isis Pharmaceuticals, Inc. Tricyclic nucleosides and oligomeric compounds prepared therefrom
US9133461B2 (en) 2012-04-10 2015-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
US9914922B2 (en) 2012-04-20 2018-03-13 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US9127274B2 (en) 2012-04-26 2015-09-08 Alnylam Pharmaceuticals, Inc. Serpinc1 iRNA compositions and methods of use thereof
US9273949B2 (en) 2012-05-11 2016-03-01 Vanderbilt University Backscattering interferometric methods
WO2013177248A2 (en) 2012-05-22 2013-11-28 Isis Pharmaceuticals, Inc. Modulation of enhancer rna mediated gene expression
EP3461895B1 (de) 2012-06-25 2020-07-01 Ionis Pharmaceuticals, Inc. Modulation der ube3a-ats-expression
US20140038182A1 (en) 2012-07-17 2014-02-06 Dna Logix, Inc. Cooperative primers, probes, and applications thereof
ES2773489T3 (es) 2012-07-27 2020-07-13 Ionis Pharmaceuticals Inc Modulación de enfermedades relacionadas con el sistema renina-angiotensina (RAS) mediante angiotensinógenos
EP2885312A4 (de) 2012-08-15 2016-01-20 Isis Pharmaceuticals Inc Verfahren zur herstellung oligomerer verbindungen unter verwendung von modifizierten kappungsprotokollen
WO2014045126A2 (en) 2012-09-18 2014-03-27 Uti Limited Partnership Treatment of pain by inhibition of usp5 de-ubiquitinase
US9175291B2 (en) 2012-10-11 2015-11-03 Isis Pharmaceuticals Inc. Modulation of androgen receptor expression
WO2014059364A1 (en) 2012-10-11 2014-04-17 Isis Pharmaceuticals, Inc. Methods of treating kennedy's disease
EP2906699A4 (de) 2012-10-11 2016-06-08 Ionis Pharmaceuticals Inc Oligomere verbindungen mit bicyclischen nukleosiden und verwendungen davon
US9029335B2 (en) 2012-10-16 2015-05-12 Isis Pharmaceuticals, Inc. Substituted 2′-thio-bicyclic nucleosides and oligomeric compounds prepared therefrom
CA2890207A1 (en) 2012-11-05 2014-05-08 Foundation Medicine, Inc. Novel ntrk1 fusion molecules and uses thereof
PT2922554T (pt) 2012-11-26 2022-06-28 Modernatx Inc Arn modificado nas porções terminais
US10272163B2 (en) 2012-12-07 2019-04-30 The Regents Of The University Of California Factor VIII mutation repair and tolerance induction
EP3434774A1 (de) 2013-01-17 2019-01-30 ModernaTX, Inc. Signal-sensor-polynukleotide zur veränderung zellulärer phänotypen
CA2898326C (en) 2013-01-18 2022-05-17 Foundation Medicine, Inc. Methods of treating cholangiocarcinoma
KR102190852B1 (ko) 2013-01-31 2020-12-14 아이오니스 파마수티컬즈, 인코포레이티드 변형된 커플링 프로토콜을 이용하는 올리고머 화합물의 제조 방법
KR20200123263A (ko) 2013-02-14 2020-10-28 아이오니스 파마수티컬즈, 인코포레이티드 지질단백질 리파제 결핍 (lpld) 모집단에서 아포지질단백질 c-iii (apociii) 발현의 조절
US20150366890A1 (en) 2013-02-25 2015-12-24 Trustees Of Boston University Compositions and methods for treating fungal infections
WO2014134179A1 (en) 2013-02-28 2014-09-04 The Board Of Regents Of The University Of Texas System Methods for classifying a cancer as susceptible to tmepai-directed therapies and treating such cancers
US20160024181A1 (en) 2013-03-13 2016-01-28 Moderna Therapeutics, Inc. Long-lived polynucleotide molecules
EP3312281A3 (de) 2013-03-14 2018-06-27 Alnylam Pharmaceuticals, Inc. Gegen komplementkomponente c5 gerichtete irna-zusammensetzungen und verfahren zur verwendung davon
US10258698B2 (en) 2013-03-14 2019-04-16 Modernatx, Inc. Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions
US8980864B2 (en) 2013-03-15 2015-03-17 Moderna Therapeutics, Inc. Compositions and methods of altering cholesterol levels
EP4286517A3 (de) 2013-04-04 2024-03-13 President and Fellows of Harvard College Therapeutische verwendungen einer genomänderung mit crispr-/cas-systemen
US10590412B2 (en) 2013-04-19 2020-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
KR102212275B1 (ko) 2013-05-01 2021-02-05 아이오니스 파마수티컬즈, 인코포레이티드 Hbv 및 ttr 발현을 조절하는 조성물 및 방법
KR102234623B1 (ko) 2013-05-22 2021-04-02 알닐람 파마슈티칼스 인코포레이티드 Ｔｍｐｒｓｓ6 조성물 및 이의 사용 방법
UY35582A (es) 2013-05-22 2014-10-31 Alnylam Pharmaceuticals Inc COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO
WO2014197835A2 (en) 2013-06-06 2014-12-11 The General Hospital Corporation Methods and compositions for the treatment of cancer
DK3007704T3 (da) 2013-06-13 2021-03-29 Antisense Therapeutics Ltd Kombinationsterapi til akromegali
EP3656386A1 (de) 2013-06-21 2020-05-27 Ionis Pharmaceuticals, Inc. Verbindungen und verfahren zur modulation der apolipoprotein-c-iii-expression zur verbesserung eines diabetischen profils
EP3730614A3 (de) 2013-07-02 2020-12-30 Ionis Pharmaceuticals, Inc. Modulatoren des wachstumshormonrezeptors
HUE056760T2 (hu) 2013-07-11 2022-03-28 Modernatx Inc A CRISPR-hez kapcsolódó fehérjéket és a szintetikus SGRNS-ket kódoló szintetikus polinukleotidokat tartalmazó készítmények és felhasználási módjaik
WO2015013673A1 (en) 2013-07-25 2015-01-29 Aurasense Therapeutics, Llc Spherical nucleic acid-based constructs as immunostimulatory agents for prophylactic and therapeutic use
EA038576B1 (ru) 2013-08-08 2021-09-16 Дзе Скриппс Рисёч Инститьют Способ сайт-специфического ферментативного мечения нуклеиновых кислот in vitro введением не встречающихся в природе нуклеотидов
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
CN105517556B (zh) 2013-08-28 2021-02-02 Ionis制药公司 前激肽释放酶(pkk)表达的调节
US20160194368A1 (en) 2013-09-03 2016-07-07 Moderna Therapeutics, Inc. Circular polynucleotides
AU2014315287A1 (en) 2013-09-03 2015-03-12 Moderna Therapeutics, Inc. Chimeric polynucleotides
ES2745758T3 (es) 2013-09-13 2020-03-03 Ionis Pharmaceuticals Inc Moduladores del factor B del complemento
US10077444B2 (en) 2013-10-02 2018-09-18 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the LECT2 gene
US10323076B2 (en) 2013-10-03 2019-06-18 Modernatx, Inc. Polynucleotides encoding low density lipoprotein receptor
AU2014331604B2 (en) 2013-10-04 2021-01-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the ALAS1 gene
US10584387B2 (en) 2013-10-09 2020-03-10 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
EP3967770B1 (de) 2013-10-21 2023-12-06 The General Hospital Corporation Verfahren im zusammenhang mit zirkulierenden tumorzellclustern und mit der behandlung von krebs
US9758546B2 (en) 2013-10-21 2017-09-12 Ionis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
US10301622B2 (en) 2013-11-04 2019-05-28 Northwestern University Quantification and spatio-temporal tracking of a target using a spherical nucleic acid (SNA)
MX2016007287A (es) 2013-12-03 2017-05-03 Univ Northwestern Particulas liposomales, metodos para elaborarlas y sus usos.
US10385388B2 (en) 2013-12-06 2019-08-20 Swift Biosciences, Inc. Cleavable competitor polynucleotides
CA2844640A1 (en) 2013-12-06 2015-06-06 The University Of British Columbia Method for treatment of castration-resistant prostate cancer
CA3107872A1 (en) 2013-12-12 2015-06-18 Alnylam Pharmaceuticals, Inc. Complement component irna compositions and methods of use thereof
EP3082840B1 (de) 2013-12-20 2021-03-24 The General Hospital Corporation Verfahren und tests im zusammenhang mit zirkulierenden tumorzellen
CN111394355A (zh) 2013-12-24 2020-07-10 Ionis制药公司 促血管生成素样3表达的调节
CA2937539A1 (en) 2014-02-04 2015-08-13 Genentech, Inc. Mutant smoothened and methods of using the same
WO2015123264A1 (en) 2014-02-11 2015-08-20 Alnylam Pharmaceuticals, Inc. Ketohexokinase (khk) irna compositions and methods of use thereof
EP3119789B1 (de) 2014-03-17 2020-04-22 Ionis Pharmaceuticals, Inc. Bicyclische carbocyclische nukleoside und daraus hergestellte oligomere verbindungen
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
IL247600B (en) 2014-03-19 2022-06-01 Ionis Pharmaceuticals Inc Preparations for the modulation of ataxin 2 expression
KR20220077933A (ko) 2014-04-01 2022-06-09 바이오젠 엠에이 인코포레이티드 Sod-1 발현을 조절하기 위한 조성물
TWI638047B (zh) 2014-04-09 2018-10-11 史基普研究協會 藉由核酸三磷酸酯轉運子將非天然或經修飾的核苷三磷酸酯輸入至細胞中
US20170044538A1 (en) 2014-04-17 2017-02-16 Biogen Ma Inc. Compositions and Methods for Modulation of SMN2 Splicing in a Subject
US10221416B2 (en) 2014-04-24 2019-03-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising alpha-beta-constrained nucleic acid
MX2016014102A (es) 2014-05-01 2017-05-03 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de similar a la angiopoyetina tipo 3.
EP3811977A1 (de) 2014-05-01 2021-04-28 Ionis Pharmaceuticals, Inc. Verfahren zur synthese von reaktiven konjugatclustern
BR112016022593B1 (pt) 2014-05-01 2022-04-26 Ionis Pharmaceuticals, Inc Compostos oligoméricos, composições que os compreendem, e usos dos mesmos
EP3137604B1 (de) 2014-05-01 2020-07-15 Ionis Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur modulierung der wachstumshormonrezeptorexpression
HUE052709T2 (hu) 2014-05-01 2021-05-28 Ionis Pharmaceuticals Inc Módosított antiszensz oligonukleotidok konjugátumai és azok alkalmazása PKK expressziójának módosítására
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
BR122020023687B1 (pt) 2014-05-22 2023-03-07 Alnylam Pharmaceuticals, Inc Agente de ácido ribonucleico (rnai) de fita dupla, seus usos e composição farmacêutica
CN106659758A (zh) 2014-06-02 2017-05-10 儿童医疗中心有限公司 用于免疫调节的组合物和方法
CN106661580B (zh) 2014-06-10 2022-02-15 鹿特丹伊拉斯谟大学医疗中心 用于治疗庞帕病的反义寡核苷酸
US20170204152A1 (en) 2014-07-16 2017-07-20 Moderna Therapeutics, Inc. Chimeric polynucleotides
US9951327B1 (en) 2014-07-17 2018-04-24 Integrated Dna Technologies, Inc. Efficient and rapid method for assembling and cloning double-stranded DNA fragments
WO2016014846A1 (en) 2014-07-23 2016-01-28 Moderna Therapeutics, Inc. Modified polynucleotides for the production of intrabodies
EP3189069A4 (de) 2014-07-31 2018-03-07 UAB Research Foundation Apoe-mimetische peptide und erhöhte wirksamkeit für klares plasmacholesterol
CN113069551A (zh) 2014-08-19 2021-07-06 西北大学 蛋白质/寡核苷酸核-壳纳米颗粒治疗剂
KR102410072B1 (ko) 2014-08-29 2022-06-20 알닐람 파마슈티칼스 인코포레이티드 트랜스타이레틴(ttr) 매개 아밀로이드증의 치료 방법
JP2017526367A (ja) 2014-08-29 2017-09-14 チルドレンズ メディカル センター コーポレーション 癌の処置のための方法および組成物
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
EP3191591A1 (de) 2014-09-12 2017-07-19 Alnylam Pharmaceuticals, Inc. Gegen komplementkomponente c5 gerichtete polynukleotidwirkstoffe und verfahren zur verwendung davon
EP3194597B1 (de) 2014-09-18 2021-06-30 The University Of British Columbia Allelspezifische therapie für morbus-huntington-haplotypen
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
WO2016061487A1 (en) 2014-10-17 2016-04-21 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
WO2016069694A2 (en) 2014-10-30 2016-05-06 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting serpinc1 (at3) and methods of use thereof
US10308937B2 (en) 2014-11-10 2019-06-04 Glaxosmithkline Intellectual Property (No. 2) Limited Combination long acting compositions and methods for Hepatitis C
MX2017006138A (es) 2014-11-10 2017-11-16 Glaxosmithkline Intellectual Property (No 2) Ltd Composiciones farmaceuticas de accion prolongada para hepatitis c.
JOP20200092A1 (ar) 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
JP2017535552A (ja) 2014-11-17 2017-11-30 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． アポリポタンパク質Ｃ３（ＡＰＯＣ３）ｉＲＮＡ組成物およびその使用方法
AU2015349680A1 (en) 2014-11-21 2017-06-08 Northwestern University The sequence-specific cellular uptake of spherical nucleic acid nanoparticle conjugates
WO2016086104A1 (en) 2014-11-25 2016-06-02 Ionis Pharmaceuticals, Inc. Modulation of ube3a-ats expression
WO2016100716A1 (en) 2014-12-18 2016-06-23 Vasant Jadhav Reversirtm compounds
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
WO2016118812A1 (en) 2015-01-23 2016-07-28 Vanderbilt University A robust interferometer and methods of using same
WO2016130806A2 (en) 2015-02-13 2016-08-18 Alnylam Pharmaceuticals, Inc. Patatin-like phospholipase domain containing 3 (pnpla3) irna compositions and methods of use thereof
US10450342B2 (en) 2015-02-23 2019-10-22 Ionis Pharmaceuticals, Inc. Method for solution phase detritylation of oligomeric compounds
AU2016222546B2 (en) 2015-02-26 2020-01-23 Ionis Pharmaceuticals, Inc. Allele specific modulators of P23H rhodopsin
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
IL302341A (en) 2015-03-27 2023-06-01 Harvard College Modified T cells and methods for their preparation and use
MX2017012610A (es) 2015-04-08 2018-03-16 Alnylam Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion del gen lect2.
KR102258516B1 (ko) 2015-04-16 2021-05-31 아이오니스 파마수티컬즈, 인코포레이티드 C9orf72 발현을 조절하기 위한 조성물
US10407678B2 (en) 2015-04-16 2019-09-10 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
WO2016201301A1 (en) 2015-06-12 2016-12-15 Alnylam Pharmaceuticals, Inc. Complement component c5 irna compositions and methods of use thereof
EP3310918B1 (de) 2015-06-18 2020-08-05 Alnylam Pharmaceuticals, Inc. Gegen hydroxysäureoxidase (glycolat-oxidase, hao1) gerichtete polynukleotidwirkstoffe und verfahren zur verwendung davon
WO2016209862A1 (en) 2015-06-23 2016-12-29 Alnylam Pharmaceuticals, Inc. Glucokinase (gck) irna compositions and methods of use thereof
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
US20180237774A1 (en) 2015-08-04 2018-08-23 Yeda Research And Development Co. Ltd. Methods of screening for riboswitches and attenuators
CA2996873A1 (en) 2015-09-02 2017-03-09 Alnylam Pharmaceuticals, Inc. Programmed cell death 1 ligand 1 (pd-l1) irna compositions and methods of use thereof
EP3353303B1 (de) 2015-09-25 2023-08-02 Ionis Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur modulation von ataxin 3-expression
KR20240033305A (ko) 2015-10-08 2024-03-12 아이오니스 파마수티컬즈, 인코포레이티드 앤지오텐시노겐 발현 조정을 위한 화합물 및 방법
CN115160439A (zh) 2015-10-30 2022-10-11 豪夫迈·罗氏有限公司 抗-HtrA1抗体及其使用方法
AU2016349954B2 (en) 2015-11-05 2022-08-25 Antisense Therapeutics Ltd Mobilizing leukemia cells
CN113952353A (zh) 2015-11-06 2022-01-21 Ionis制药公司 调节载脂蛋白(a)表达
US11058709B1 (en) 2015-12-04 2021-07-13 Ionis Pharmaceuticals, Inc. Methods of treating breast cancer
US10993995B2 (en) 2015-12-07 2021-05-04 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
WO2017120365A1 (en) 2016-01-05 2017-07-13 Ionis Pharmaceuticals, Inc. Methods for reducing lrrk2 expression
EP3408649B1 (de) 2016-01-29 2023-06-14 Vanderbilt University Interferometrie mit antwortfunktion in freier lösung
AU2017213826A1 (en) 2016-02-04 2018-08-23 Curis, Inc. Mutant smoothened and methods of using the same
AU2017234678A1 (en) 2016-03-16 2018-08-16 Ionis Pharmaceuticals, Inc. Methods of modulating KEAP1
WO2017161168A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Modulation of dyrk1b expression
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
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
EP3471781A4 (de) 2016-06-17 2020-05-06 Ionis Pharmaceuticals, Inc. Modulierung der gys1-expression
DK3475295T3 (da) 2016-06-24 2022-10-24 Scripps Research Inst Hidtil ukendt nukleosidtriphosphat-transportør og anvendelser deraf
WO2018015936A2 (en) 2016-07-21 2018-01-25 Maxcyte, Inc. Methods and compositions for modifying genomic dna
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
US11364304B2 (en) 2016-08-25 2022-06-21 Northwestern University Crosslinked micellar spherical nucleic acids
WO2018055577A1 (en) 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
US11400161B2 (en) 2016-10-06 2022-08-02 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
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
WO2018102745A1 (en) 2016-12-02 2018-06-07 Cold Spring Harbor Laboratory Modulation of lnc05 expression
JP7058656B2 (ja) 2016-12-16 2022-04-22 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 トランスサイレチン（ＴＴＲ）ｉＲＮＡ組成物を用いてＴＴＲ関連疾患を治療または予防するための方法
SG11201906735RA (en) 2017-01-23 2019-08-27 Regeneron Pharma Hydroxysteroid 17-beta dehydrogenase 13 (hsd17b13) variants and uses thereof
WO2018165564A1 (en) 2017-03-09 2018-09-13 Ionis Pharmaceuticals, Inc. Morpholino modified oligomeric compounds
WO2018183969A1 (en) 2017-03-30 2018-10-04 California Institute Of Technology Barcoded rapid assay platform for efficient analysis of candidate molecules and methods of making and using the platform
AU2018254437A1 (en) 2017-04-18 2019-11-28 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis B virus (HBV) infection
WO2018193428A1 (en) 2017-04-20 2018-10-25 Synthena Ag Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
KR20190141184A (ko) 2017-04-20 2019-12-23 신테나 아게 트리시클로-dna 뉴클레오시드를 포함하는 변형 올리고머 화합물 및 그의 용도
US11433131B2 (en) 2017-05-11 2022-09-06 Northwestern University Adoptive cell therapy using spherical nucleic acids (SNAs)
AU2018300069A1 (en) 2017-07-11 2020-02-27 Synthorx, Inc. Incorporation of unnatural nucleotides and methods thereof
AU2018301477A1 (en) 2017-07-13 2020-02-27 Alnylam Pharmaceuticals Inc. Lactate dehydrogenase a (LDHA) iRNA compositions and methods of use thereof
MX2020000387A (es) 2017-07-13 2020-08-17 Univ Northwestern Método general y directo para preparar nanopartículas de estructura organometálica funcionalizadas con oligonucleotidos.
US20200181220A1 (en) 2017-08-03 2020-06-11 Synthorx, Inc. Cytokine conjugates for the treatment of proliferative and infectious diseases
US11197884B2 (en) 2017-08-18 2021-12-14 Ionis Pharmaceuticals, Inc. Modulation of the notch signaling pathway for treatment of respiratory disorders
WO2019051173A1 (en) 2017-09-08 2019-03-14 Ionis Pharmaceuticals, Inc. MODULATORS OF SMAD7 EXPRESSION
US11806360B2 (en) 2017-09-19 2023-11-07 Alnylam Pharmaceuticals, Inc. Compositions and methods for treating transthyretin (TTR) mediated amyloidosis
WO2019089922A1 (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
EP3710587A1 (de) 2017-11-16 2020-09-23 Alnylam Pharmaceuticals, Inc. Apolipoprotein-c3 kisspeptin 1 (kiss1)-irna-zusammensetzungen und verfahren zur verwendung davon
WO2019100039A1 (en) 2017-11-20 2019-05-23 Alnylam Pharmaceuticals, Inc. Serum amyloid p component (apcs) irna compositions and methods of use thereof
MX2020006012A (es) 2017-12-18 2020-09-14 Alnylam Pharmaceuticals Inc Composiciones de arni de la caja 1 del grupo de alta movilidad (hmgb1) y métodos de uso de las mismas.
WO2019126641A2 (en) 2017-12-21 2019-06-27 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
TW201934129A (zh) 2018-01-15 2019-09-01 美商Ionis製藥公司 Ｄｎｍ２表現之調節劑
US20190233816A1 (en) 2018-01-26 2019-08-01 Massachusetts Institute Of Technology Structure-guided chemical modification of guide rna and its applications
KR20200127207A (ko) 2018-02-26 2020-11-10 신톡스, 인크. Il-15 접합체 및 이의 용도
TW202000199A (zh) 2018-03-02 2020-01-01 美商Ionis製藥公司 Irf4 表現之調節劑
US11732260B2 (en) 2018-03-02 2023-08-22 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-β precursor protein
US11661601B2 (en) 2018-03-22 2023-05-30 Ionis Pharmaceuticals, Inc. Methods for modulating FMR1 expression
EP3772928A4 (de) 2018-04-06 2021-12-29 Children's Medical Center Corporation Zusammensetzungen und verfahren zur prägung der umprogrammierung und modulation von somatischen zellen
MX2020010721A (es) 2018-04-11 2020-11-06 Ionis Pharmaceuticals Inc Moduladores de la expresion de ezh2.
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
MX2020011911A (es) 2018-05-09 2021-01-29 Ionis Pharmaceuticals Inc Compuestos y metodos para reducir de la expresion de atxn3.
US20210355497A1 (en) 2018-05-09 2021-11-18 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing fxi expression
TW202016304A (zh) 2018-05-14 2020-05-01 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
CA3103429A1 (en) 2018-06-14 2019-12-19 Don W. Cleveland Compounds and methods for increasing stmn2 expression
US11332746B1 (en) 2018-06-27 2022-05-17 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing LRRK2 expression
AR115847A1 (es) 2018-07-25 2021-03-03 Ionis Pharmaceuticals Inc Compuestos y métodos para reducir la expresión de la atxn2
CN112673103A (zh) 2018-08-13 2021-04-16 阿尔尼拉姆医药品有限公司 乙型肝炎病毒(HBV)dsRNA剂组合物及其使用方法
US20210348162A1 (en) 2018-08-16 2021-11-11 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
JP2022500442A (ja) 2018-09-14 2022-01-04 ノースウェスタン ユニバーシティ Ｄｎａとのタンパク質重合のプログラミング
JP2022500003A (ja) 2018-09-18 2022-01-04 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. ケトヘキソキナーゼ（ＫＨＫ）ｉＲＮＡ組成物およびその使用方法
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
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
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
CN113646430A (zh) 2018-11-15 2021-11-12 Ionis制药公司 Irf5表达的调节剂
US20210332495A1 (en) 2018-12-06 2021-10-28 Northwestern University Protein Crystal Engineering Through DNA Hybridization Interactions
JP2022515744A (ja) 2018-12-20 2022-02-22 プラクシス プレシジョン メディシンズ， インコーポレイテッド Ｋｃｎｔ１関連障害の治療のための組成物及び方法
JP2022515778A (ja) 2018-12-20 2022-02-22 ヴィア・バイオテクノロジー・インコーポレイテッド 併用ｈｂｖ療法
MX2021008628A (es) 2019-01-16 2021-11-17 Genzyme Corp Composiciones de arni para serpinc1 y metodos de uso de las mismas.
CA3128093A1 (en) 2019-01-31 2020-08-06 Ionis Pharmaceuticals, Inc. Modulators of yap1 expression
MA54952A (fr) 2019-02-06 2022-05-11 Synthorx Inc Conjugués d'il-2 et méthodes d'utilisation de ceux-ci
AU2020227824A1 (en) 2019-02-27 2021-08-26 Ionis Pharmaceuticals, Inc. Modulators of MALAT1 expression
CN117431244A (zh) 2019-03-29 2024-01-23 Ionis制药公司 用于调节ube3a-ats的化合物和方法
JPWO2020203880A1 (de) 2019-03-29 2020-10-08
CN114126628A (zh) 2019-05-13 2022-03-01 维尔生物科技有限公司 用于治疗乙型肝炎病毒(hbv)感染的组合物及方法
WO2020236600A1 (en) 2019-05-17 2020-11-26 Alnylam Pharmaceuticals, Inc. Oral delivery of oligonucleotides
EP3983543A4 (de) 2019-06-14 2023-05-03 The Scripps Research Institute Reagenzien und verfahren zur replikation, transkription und translation in halbsynthetischen organismen
US11786546B2 (en) 2019-07-26 2023-10-17 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating GFAP
EP4007811A2 (de) 2019-08-01 2022-06-08 Alnylam Pharmaceuticals, Inc. Carboxypeptidase-b2(cpb2)-irna-zusammensetzungen und verfahren zur verwendung davon
WO2021022109A1 (en) 2019-08-01 2021-02-04 Alnylam Pharmaceuticals, Inc. SERPIN FAMILY F MEMBER 2 (SERPINF2) 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
CA3150163A1 (en) 2019-08-15 2021-02-18 Synthorx, Inc. Immuno oncology combination therapies with il-2 conjugates
WO2021030778A1 (en) 2019-08-15 2021-02-18 Ionis Pharmaceuticals, Inc. Linkage modified oligomeric compounds and uses thereof
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
US20220290152A1 (en) 2019-09-03 2022-09-15 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of the lect2 gene
BR112022003335A2 (pt) 2019-09-10 2022-05-24 Synthorx Inc Conjugados de il-2 e métodos de uso para tratar doenças autoimunes
US20220389429A1 (en) 2019-10-04 2022-12-08 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing ugt1a1 gene expression
EP4045652A1 (de) 2019-10-18 2022-08-24 Alnylam Pharmaceuticals, Inc. Irna-zusammensetzungen eines elements der solute-carrier-familie und verwendungsverfahren dafür
KR20220084399A (ko) 2019-10-22 2022-06-21 알닐람 파마슈티칼스 인코포레이티드 보체 성분 C3 iRNA 조성물 및 이의 사용 방법
AR120341A1 (es) 2019-11-01 2022-02-09 Alnylam Pharmaceuticals Inc COMPOSICIONES DE AGENTES DE ARNi CONTRA LA HUNTINGTINA (HTT) Y SUS MÉTODOS DE USO
EP4051796A1 (de) 2019-11-01 2022-09-07 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zum unterdrücken der dnajb1-prkaca-fusionsgenexpression
AU2020380275A1 (en) 2019-11-04 2022-04-14 Synthorx, Inc. Interleukin 10 conjugates and uses thereof
EP4055165A1 (de) 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Transthyretin (ttr)-irna-zusammensetzungen und verfahren zur verwendung davon für die behandlung oder prävention ttr-assoziierter augenerkrankungen
BR112022007795A2 (pt) 2019-11-06 2022-07-05 Alnylam Pharmaceuticals Inc Administração extra-hepática
EP4061945A1 (de) 2019-11-22 2022-09-28 Alnylam Pharmaceuticals, Inc. Ataxin3 (atxn3)-rnai-wirkstoffzusammensetzungen und verfahren zu deren verwendung
JP2023506181A (ja) 2019-12-13 2023-02-15 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 ヒト第９染色体オープンリーディングフレーム７２（Ｃ９ＯＲＦ７２）ｉＲＮＡ剤組成物およびその使用方法
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
US20230057461A1 (en) 2020-01-27 2023-02-23 The U.S.A., 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)
CA3170377A1 (en) 2020-02-10 2021-08-19 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing vegf-a expression
JP2023514336A (ja) 2020-02-18 2023-04-05 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 アポリポタンパク質Ｃ３（ＡＰＯＣ３）ｉＲＮＡ組成物およびその使用法
AU2021225957A1 (en) 2020-02-28 2022-09-08 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating SMN2
EP4114947A1 (de) 2020-03-05 2023-01-11 Alnylam Pharmaceuticals, Inc. Komplementkomponenten-c3-irna-zusammensetzungen und verfahren zur verwendung davon zur behandlung oder prävention von mit komplementkomponenten-c3-assoziierten erkrankungen
JP2023516095A (ja) 2020-03-06 2023-04-17 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 ケトヘキソキナーゼ（ＫＨＫ）ｉＲＮＡ組成物およびその使用方法
EP4121534A1 (de) 2020-03-18 2023-01-25 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur behandlung von patienten mit einer heterozygoten alaninglyoxylat-aminotransferase-genvariante (agxt)
WO2021195307A1 (en) 2020-03-26 2021-09-30 Alnylam Pharmaceuticals, Inc. Coronavirus irna compositions and methods of use thereof
EP4127171A2 (de) 2020-03-30 2023-02-08 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur ausschaltung der dnajc15-genexpression
AR121769A1 (es) 2020-04-06 2022-07-06 Alnylam Pharmaceuticals Inc Composiciones y métodos para el silenciamiento de la expresión de myoc
WO2021206922A1 (en) 2020-04-07 2021-10-14 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
EP4133076A1 (de) 2020-04-07 2023-02-15 Alnylam Pharmaceuticals, Inc. Irna-zusammensetzungen aus angiotensin-konvertierendem enzym 2 (ace2) und verfahren zur verwendung davon
IL297702A (en) 2020-04-27 2022-12-01 Alnylam Pharmaceuticals Inc Compositions of apolipoprotein e (apoe) RNA material and methods of using them
CA3181198A1 (en) 2020-04-30 2021-11-04 Alnylam Pharmaceuticals, Inc. Complement factor b (cfb) irna compositions and methods of use thereof
CA3181546A1 (en) 2020-05-01 2021-11-04 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating atxn1
CN115667513A (zh) 2020-05-12 2023-01-31 田边三菱制药株式会社 用于调节Ataxin 3表达的化合物、方法和药物组合物
EP4150078A1 (de) 2020-05-15 2023-03-22 Korro Bio, Inc. Verfahren und zusammensetzungen zur adar-vermittelten editierung von argininosuccinat-lyase (asl)
WO2021231692A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of otoferlin (otof)
WO2021231685A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of transmembrane channel-like protein 1 (tmc1)
WO2021231691A1 (en) 2020-05-15 2021-11-18 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of retinoschisin 1 (rsi)
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)
CA3162416C (en) 2020-05-15 2023-07-04 Korro Bio, Inc. Methods and compositions for the adar-mediated editing of argininosuccinate synthetase (ass1)
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)
EP4150086A1 (de) 2020-05-15 2023-03-22 Korro Bio, Inc. Verfahren und zusammensetzungen zur adar-vermittelten bearbeitung von leucinreicher repeat-kinase 2 (lrrk2)
EP4153746A1 (de) 2020-05-21 2023-03-29 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur hemmung der marc1-genexpression
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 (de) 2020-06-09 2023-04-12 Alnylam Pharmaceuticals, Inc. Rnai-zusammensetzungen und verfahren zur verwendung davon zur verabreichung durch inhalation
WO2021257782A1 (en) 2020-06-18 2021-12-23 Alnylam Pharmaceuticals, Inc. XANTHINE DEHYDROGENASE (XDH) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EP4171747A1 (de) 2020-06-24 2023-05-03 VIR Biotechnology, Inc. Manipulierte hepatitis-b-virus-neutralisierende antikörper und verwendungen davon
BR112022026236A2 (pt) 2020-06-25 2023-01-17 Synthorx Inc Terapia de combinação de imuno-oncologia com conjugados de il-2 e anticorpos anti-egfr
EP4172338A2 (de) 2020-06-29 2023-05-03 Ionis Pharmaceuticals, Inc. Verbindungen und verfahren zur modulierung von plp1
US20230257745A1 (en) 2020-07-10 2023-08-17 Alnylam Pharmaceuticals, Inc. Circular siRNAs
EP4217489A1 (de) 2020-09-24 2023-08-02 Alnylam Pharmaceuticals, Inc. Dipeptidylpeptidase 4 (dpp4)-irna-zusammensetzungen und verfahren zur verwendung davon
EP3978608A1 (de) 2020-10-05 2022-04-06 SQY Therapeutics Oligomere verbindung zur dystrophinrettung in dmd-patienten während des skipping exon-51
EP4225917A1 (de) 2020-10-05 2023-08-16 Alnylam Pharmaceuticals, Inc. G-protein-gekoppelter rezeptor-75 (gpr75)-irna-zusammensetzungen und verfahren zur verwendung davon
BR112023006024A2 (pt) 2020-10-09 2023-05-09 Synthorx Inc Terapias de imuno-oncologia com conjugados de il-2
WO2022076853A1 (en) 2020-10-09 2022-04-14 Synthorx, Inc. Immuno oncology combination therapy with il-2 conjugates and pembrolizumab
CA3198823A1 (en) 2020-10-21 2022-04-28 Alnylam Pharmaceuticals, Inc. Methods and compositions for treating primary hyperoxaluria
WO2022087329A1 (en) 2020-10-23 2022-04-28 Alnylam Pharmaceuticals, Inc. Mucin 5b (muc5b) irna compositions and methods of use thereof
CA3200595A1 (en) 2020-11-13 2022-05-19 Alnylam Pharmaceuticals, Inc. Coagulation factor v (f5) irna compositions and methods of use thereof
IL302817A (en) 2020-11-18 2023-07-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating angiotensinogen expression
CA3201452A1 (en) 2020-12-01 2022-06-09 Alnylam Pharmaceuticals, Inc. Methods and compositions for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
WO2022125490A1 (en) 2020-12-08 2022-06-16 Alnylam Pharmaceuticals, Inc. Coagulation factor x (f10) irna compositions and methods of use thereof
JP2024501288A (ja) 2020-12-23 2024-01-11 フラッグシップ パイオニアリング イノベーションズ シックス，エルエルシー 修飾ｔｒｅｍの組成物及びその使用
AU2021411579A1 (en) 2020-12-31 2023-07-13 Alnylam Pharmaceuticals, Inc. Cyclic-disulfide modified phosphate based oligonucleotide prodrugs
EP4271695A2 (de) 2020-12-31 2023-11-08 Alnylam Pharmaceuticals, Inc. Auf 2'-modifiziertem nukleosid basierende oligonukleotid-prodrugs
EP4274896A1 (de) 2021-01-05 2023-11-15 Alnylam Pharmaceuticals, Inc. Komplementkomponente 9 (c9)-irna-zusammensetzungen und verfahren zur verwendung davon
EP4291243A1 (de) 2021-02-12 2023-12-20 Synthorx, Inc. Lungenkrebskombinationstherapie mit il-2-konjugaten und einem anti-pd-1-antikörper oder antigenbindendem fragment davon
IL304880A (en) 2021-02-12 2023-10-01 Alnylam Pharmaceuticals Inc Superoxide dismutase 1 (SOD1) IRNA compositions and methods of using them to treat or prevent superoxide dismutase 1- (SOD1-) associated neurodegenerative diseases
TW202245843A (zh) 2021-02-12 2022-12-01 美商欣爍克斯公司 Ｉｌ－２接合物及西米普利單抗（ｃｅｍｉｐｌｉｍａｂ）之皮膚癌組合療法
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
IL305414A (en) 2021-03-04 2023-10-01 Alnylam Pharmaceuticals Inc Angiopoietin-like 3 (ANGPTL3) IRNA compositions and methods of using them
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
JP2024512635A (ja) 2021-03-29 2024-03-19 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 ハンチンチン（ＨＴＴ）ｉＲＮＡ剤組成物およびその使用方法
IL307298A (en) 2021-03-31 2023-11-01 Entrada Therapeutics Inc Cell-penetrating circular peptides
WO2022212153A1 (en) 2021-04-01 2022-10-06 Alnylam Pharmaceuticals, Inc. Proline dehydrogenase 2 (prodh2) irna compositions and methods of use thereof
KR20240001207A (ko) 2021-04-26 2024-01-03 알닐람 파마슈티칼스 인코포레이티드 막관통 프로테아제, 세린 6(TMPRSS6) iRNA 조성물 및 이의 사용 방법
WO2022232343A1 (en) 2021-04-29 2022-11-03 Alnylam Pharmaceuticals, Inc. Signal transducer and activator of transcription factor 6 (stat6) irna compositions and methods of use thereof
EP4334448A1 (de) 2021-05-03 2024-03-13 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur behandlung von transthyretin(ttr)-vermittelter amyloidose
IL308353A (en) 2021-05-10 2024-01-01 Entrada Therapeutics Inc Compositions and methods for modulating tissue distribution of intracellular therapies
WO2022240721A1 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. Compositions and methods for modulating interferon regulatory factor-5 (irf-5) activity
WO2022240760A2 (en) 2021-05-10 2022-11-17 Entrada Therapeutics, Inc. COMPOSITIONS AND METHODS FOR MODULATING mRNA SPLICING
EP4341401A1 (de) 2021-05-18 2024-03-27 Alnylam Pharmaceuticals, Inc. Natriumglucose-cotransporter-2-(sglt2)-irna-zusammensetzungen und verfahren zur verwendung davon
EP4341405A1 (de) 2021-05-20 2024-03-27 Korro Bio, Inc. Verfahren und zusammensetzungen für adar-vermittelte bearbeitung
WO2022256283A2 (en) 2021-06-01 2022-12-08 Korro Bio, Inc. Methods for restoring protein function using adar
TW202317762A (zh) 2021-06-02 2023-05-01 美商艾拉倫製藥股份有限公司 含有類ＰＡＴＡＴＩＮ磷脂酶結構域３（ＰＮＰＬＡ３）的ｉＲＮＡ組成物及其使用方法
WO2022256534A1 (en) 2021-06-03 2022-12-08 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
AR126000A1 (es) 2021-06-04 2023-08-30 Alnylam Pharmaceuticals Inc Agentes de arni del marco de lectura abierto 72 del cromosoma 9 humano (c9orf72), composiciones y métodos de uso de los mismos
EP4351541A2 (de) 2021-06-08 2024-04-17 Alnylam Pharmaceuticals, Inc. Zusammensetzungen und verfahren zur behandlung oder prävention von erkrankungen im zusammenhang mit morbus stargardt und/oder retinal-bindendem protein 4 (rbp4)
CA3223192A1 (en) 2021-06-18 2022-12-22 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ifnar1 expression
KR20240038967A (ko) 2021-06-23 2024-03-26 엔트라다 테라퓨틱스, 인크. Cug 반복을 표적화하기 위한 안티센스 화합물 및 방법
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
WO2023283403A2 (en) 2021-07-09 2023-01-12 Alnylam Pharmaceuticals, Inc. Bis-rnai compounds for cns delivery
TW202333748A (zh) 2021-07-19 2023-09-01 美商艾拉倫製藥股份有限公司 用於處置具有或有風險發展非原發性高草酸鹽尿疾病或病症的個體的方法及組成物
IL309897A (en) 2021-07-21 2024-03-01 Alnylam Pharmaceuticals Inc Target gene IRNA compositions associated with a metabolic disorder and methods of using them
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
TW202328445A (zh) 2021-08-03 2023-07-16 美商艾拉倫製藥股份有限公司 甲狀腺素運載蛋白（ＴＴＲ）ｉＲＮＡ組成物及其使用方法
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
CA3229661A1 (en) 2021-09-01 2023-03-09 Xiang Li Compounds and methods for skipping exon 44 in duchenne muscular dystrophy
WO2023044370A2 (en) 2021-09-17 2023-03-23 Alnylam Pharmaceuticals, Inc. Irna compositions and methods for silencing complement component 3 (c3)
AU2022345881A1 (en) 2021-09-20 2024-03-21 Alnylam Pharmaceuticals, Inc. Inhibin subunit beta e (inhbe) modulator compositions and methods of use thereof
WO2023064530A1 (en) 2021-10-15 2023-04-20 Alnylam Pharmaceuticals, Inc. Extra-hepatic delivery irna compositions and methods of use thereof
WO2023069603A1 (en) 2021-10-22 2023-04-27 Korro Bio, Inc. Methods and compositions for disrupting nrf2-keap1 protein interaction by adar mediated rna editing
WO2023076450A2 (en) 2021-10-29 2023-05-04 Alnylam Pharmaceuticals, Inc. HUNTINGTIN (HTT) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
TW202333749A (zh) 2021-10-29 2023-09-01 美商艾拉倫製藥股份有限公司 補體因子ｂ（ｃｆｂ）ｉｒｎａ組成物及其使用方法
WO2023092060A1 (en) 2021-11-18 2023-05-25 Cornell University Microrna-dependent mrna switches for tissue-specific mrna-based therapies
WO2023122573A1 (en) 2021-12-20 2023-06-29 Synthorx, Inc. Head and neck cancer combination therapy comprising an il-2 conjugate and pembrolizumab
WO2023122750A1 (en) 2021-12-23 2023-06-29 Synthorx, Inc. Cancer combination therapy with il-2 conjugates and cetuximab
WO2023141314A2 (en) 2022-01-24 2023-07-27 Alnylam Pharmaceuticals, Inc. Heparin sulfate biosynthesis pathway enzyme irna agent compositions and methods of use thereof
WO2023220744A2 (en) 2022-05-13 2023-11-16 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
WO2024006999A2 (en) 2022-06-30 2024-01-04 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
WO2024059165A1 (en) 2022-09-15 2024-03-21 Alnylam Pharmaceuticals, Inc. 17b-hydroxysteroid dehydrogenase type 13 (hsd17b13) irna compositions and methods of use thereof
WO2024073732A1 (en) 2022-09-30 2024-04-04 Alnylam Pharmaceuticals, Inc. Modified double-stranded rna agents

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IT1196261B (it) * 1984-09-20 1988-11-16 Pierrel Spa Derivati nucleosidici e purinici 8-sostituiti
US4965270A (en) * 1987-05-30 1990-10-23 Beecham Group P.L.C. Purine derivatives
GB8724765D0 (en) * 1987-10-22 1987-11-25 Beecham Group Plc Process
EP0353955A3 (de) * 1988-08-02 1991-08-14 Beecham Group Plc Chemische Verbindungen
GB8918827D0 (en) * 1989-08-17 1989-09-27 Beecham Group Plc Novel compounds
IT1248528B (it) * 1991-06-21 1995-01-19 Pierrel Spa Derivati di eteri e tioeteri (etero) aromatici aventi attivita` antiiperlipidemica, procedimento per la loro preparazione e composizioni farmaceutiche che li contengono.

Also Published As

Publication number Publication date
WO1994022864A1 (en) 1994-10-13
US5576427A (en) 1996-11-19
ES2107205T3 (es) 1997-11-16
EP0691968A1 (de) 1996-01-17
EP0691968B1 (de) 1997-07-16
JPH08508492A (ja) 1996-09-10
CA2159631A1 (en) 1994-10-13
AU6449394A (en) 1994-10-24
ATE155467T1 (de) 1997-08-15
GR3025053T3 (en) 1998-01-30
DE69404289D1 (de) 1997-08-21
DK0691968T3 (da) 1998-02-23

Similar Documents

Publication Publication Date Title
DE69404289D1 (de) Acyclische nucleosid analoge und sie enthaltende oligonucleotidsequenzen
ES2382807T3 (es) Nuevos ácidos nucleicos artificiales del tipo de enlace N-O con reticulación
KR950032263A (ko) 2'-에테르 그룹을 갖는 뉴클레오사이드 및 올리고 뉴클레오타이드
IL110091A (en) POLYSUBSTITUTED IMIDAZO £4, 5-c| PYRIDIN-4-AMINES AND PHARMACEUTICAL COMPOSITIONS CONTAINING THEM
HU9500503D0 (en) Derivatives of valproic and 2-valproenoic acid amides and use anticonvulsants
NO20012411L (no) Pyrrolidin-derivater-CCR-3-reseptorantagonister
ES2128535T3 (es) Nucleosidos y oligonucleotidos con grupos 2'-eter.
CN102918156A (zh) 用于控制基因表达的单链核酸分子
GB9803226D0 (en) Chemical compounds
HU9603185D0 (en) New azolidinediones as antihyperglycemic agents
HUP9901065A2 (hu) Új barbitursavszármazékok, ezen vegyületeket tartalmazó gyógyászati készítmények és eljárás előállításukra
IL115746A0 (en) Polyanionic benzylglycosides and pharmaceutical compositions containing them
DE69107959T2 (de) 3-Cycloalkylpropansäureamide, ihre Tautomere und Salze, Verfahren zu ihrer Herstellung, ihre Verwendung als Arzneimittel und sie enthaltende Zusammensetzungen.
TW427982B (en) Polysubstituted 3-acylamino-5-phenyl-1,4-benzodiazepin-2-one derivatives, process for their preparation and the pharmaceutical compositions containing them
GB2269589B (en) 3-Hydroxy-pyridin-4-ones and their use in pharmaceutical compositions
ES2166081T3 (es) Derivados de nucleosidos con grupos protectores fotolabiles.
PL319677A1 (en) Method of obtaining p-chiral analoques of nucleotides
TW324007B (en) N-substituted diazabicycloheptane derivative and its pharmaceutical use
GR3030486T3 (en) Pyrrolo-pyridazines with gastro-intestinal protective action.
GB8907799D0 (en) Heteroaryl-3-oxo-propanenitrile derivatives useful in the treatment of rheumatoid arthritis and other autoimmune diseases
HU9200069D0 (en) Process for producing 1,4-dihydro-pyridines and for producing pharmaceutical preparatives containing said compounds as active substance
BR9604907B1 (pt) derivados de nucleosidios homocarbociclico de pirimidinodiona substituìda antiviral e composição contendo derivados de nucleosidios homocarbociclico de pirimidinodiona substituìda antiviral com princìpios ativos.
HUP9701325A1 (hu) Új 2,3-benzodiazepin-származékok
ES2109177A1 (es) Procedimiento general de preparacion de oligonucleotidos ciclicos e intermedios para el mismo.
DE69411380D1 (de) Neue heterocyclische acyldipeptide, verfahren zu deren herstellung und diese enthaltende pharmazeutische zusammensetzung

Legal Events

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