ES2640091T3 - Compuestos de oligonucleótidos que comprenden excedentes no nucleótidos - Google Patents

Compuestos de oligonucleótidos que comprenden excedentes no nucleótidos Download PDF

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Publication number
ES2640091T3
ES2640091T3 ES11701706.1T ES11701706T ES2640091T3 ES 2640091 T3 ES2640091 T3 ES 2640091T3 ES 11701706 T ES11701706 T ES 11701706T ES 2640091 T3 ES2640091 T3 ES 2640091T3
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structural unit
nucleotide
phosphate
c3pi
terminal
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Spanish (es)
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Sharon Avkin-Nachum
Elena Feinstein
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Quark Pharmaceuticals Inc
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Quark Pharmaceuticals Inc
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
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    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
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    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/317Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12N2310/3212'-O-R Modification
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/33Chemical structure of the base
    • C12N2310/332Abasic residue
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3527Other alkyl chain

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ES11701706.1T 2010-01-07 2011-01-06 Compuestos de oligonucleótidos que comprenden excedentes no nucleótidos Active ES2640091T3 (es)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US29287810P 2010-01-07 2010-01-07
US292878P 2010-01-07
WOPCT/US2010/049047 2010-09-16
PCT/US2010/049047 WO2011084193A1 (en) 2010-01-07 2010-09-16 Oligonucleotide compounds comprising non-nucleotide overhangs
PCT/US2011/020298 WO2011085056A1 (en) 2010-01-07 2011-01-06 Oligonucleotide compounds comprising non-nucleotide overhangs

Publications (1)

Publication Number Publication Date
ES2640091T3 true ES2640091T3 (es) 2017-10-31

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ES11701706.1T Active ES2640091T3 (es) 2010-01-07 2011-01-06 Compuestos de oligonucleótidos que comprenden excedentes no nucleótidos

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US (2) US8859751B2 (enExample)
EP (1) EP2521783B1 (enExample)
JP (1) JP5824463B2 (enExample)
CN (1) CN102770541B (enExample)
ES (1) ES2640091T3 (enExample)
WO (2) WO2011084193A1 (enExample)

Families Citing this family (37)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
TWI611021B (zh) 2009-12-09 2018-01-11 日東電工股份有限公司 Hsp47表現之調節
NZ604094A (en) * 2010-06-24 2014-11-28 Quark Pharmaceuticals Inc Double stranded rna compounds to rhoa and use thereof
ES2930555T3 (es) 2010-10-22 2022-12-16 Olix Pharmaceuticals Inc Moléculas de ácido nucleico que inducen la interferencia de ARN y usos de las mismas
AU2011338682B2 (en) 2010-12-06 2017-04-27 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising threose modifications
WO2012118911A1 (en) 2011-03-03 2012-09-07 Quark Pharmaceuticals, Inc. Oligonucleotide modulators of the toll-like receptor pathway
US9796979B2 (en) 2011-03-03 2017-10-24 Quark Pharmaceuticals Inc. Oligonucleotide modulators of the toll-like receptor pathway
BR112014010769B1 (pt) 2011-11-03 2021-08-03 Quark Pharmaceuticals, Inc Uso de um composto de rna de fita dupla para preparar um medicamento para tratar doença de ménière
WO2013070821A1 (en) 2011-11-08 2013-05-16 Quark Pharmaceuticals, Inc. Methods and compositions for treating diseases, disorders or injury of the nervous system
JP2015509085A (ja) 2012-01-01 2015-03-26 キュービーアイ エンタープライゼズ リミテッドQbi Enterprises Ltd. 治療剤および診断剤の選択的送達のためのendo180を標的とする粒子
EP2800812A1 (en) 2012-01-04 2014-11-12 Quark Pharmaceuticals, Inc. Double-stranded rna compounds to casp2 and uses thereof
US9707235B1 (en) 2012-01-13 2017-07-18 University Of Kentucky Research Foundation Protection of cells from degeneration and treatment of geographic atrophy
EP3514236A1 (en) 2012-05-22 2019-07-24 Olix Pharmaceuticals, Inc. Rna-interference-inducing nucleic acid molecule able to penetrate into cells, and use therefor
WO2014043292A1 (en) 2012-09-12 2014-03-20 Quark Pharmaceuticals, Inc. Double-stranded oligonucleotide molecules to p53 and methods of use thereof
DK2895607T3 (da) * 2012-09-12 2021-05-25 Quark Pharmaceuticals Inc Dobbeltstrengede oligonukleotidmolekyler til ddit4 og fremgangsmåder til anvendelse deraf
JP6346568B2 (ja) * 2012-11-29 2018-06-20 国立大学法人岐阜大学 Rna干渉剤、その製造方法及びその利用
US9889200B2 (en) 2013-07-31 2018-02-13 Qbi Enterprises Ltd. Sphingolipid-polyalkylamine-oligonucleotide compounds
WO2015015498A1 (en) 2013-07-31 2015-02-05 Qbi Enterprises Ltd. Methods of use of sphingolipid polyalkylamine oligonucleotide compounds
KR101696704B1 (ko) 2013-12-17 2017-01-16 주식회사 인코드젠 오프-타겟을 막기 위해 변형된 rna 간섭 유도 핵산 및 그 용도
DK3444349T3 (da) 2014-05-29 2021-04-12 Quark Pharmaceuticals Inc Fremgangsmåder og sammensætninger til forebyggelse af iskæmsik reperfusionslæsion i organer
WO2016149020A1 (en) * 2015-03-17 2016-09-22 Arrowhead Research Corporation Rna interference agents
PL3350333T3 (pl) 2015-09-17 2022-03-07 Modernatx, Inc. Polinukleotydy zawierające region stabilizujący ogon
CN108699556B (zh) 2015-11-16 2023-02-17 奥利克斯医药有限公司 使用靶向myd88或tlr3的rna复合物治疗老年性黄斑变性
CN108779464B (zh) 2016-02-02 2022-05-17 奥利克斯医药有限公司 使用靶向angpt2和pdgfb的rna复合物治疗血管生成相关性疾病
KR102825946B1 (ko) 2016-02-02 2025-06-27 올릭스 주식회사 IL4Rα, TRPA1, 또는 F2RL1을 표적화하는 RNA 복합체를 사용한 아토피 피부염 및 천식의 치료
US10301628B2 (en) 2016-04-11 2019-05-28 Olix Pharmaceuticals, Inc. Treatment of idiopathic pulmonary fibrosis using RNA complexes that target connective tissue growth factor
KR101916652B1 (ko) 2016-06-29 2018-11-08 올릭스 주식회사 작은 간섭 rna의 rna 간섭효과 증진용 화합물 및 이의 용도
AU2017368050A1 (en) 2016-11-29 2019-06-20 Puretech Lyt, Inc. Exosomes for delivery of therapeutic agents
KR20190108167A (ko) 2017-02-10 2019-09-23 성균관대학교산학협력단 Rna 간섭을 위한 긴 이중가닥 rna
WO2020191177A1 (en) * 2019-03-21 2020-09-24 Sudhir Agrawal Antisense oligonucleotides for allele specificity
KR102421750B1 (ko) * 2020-05-26 2022-07-19 올릭스 주식회사 MyD88을 표적으로 하는 RNAi 제제 및 이의 용도
WO2022162155A1 (en) * 2021-01-30 2022-08-04 E-Therapeutics Plc Nucleic acids containing abasic nucleotides
WO2023059948A1 (en) * 2021-10-08 2023-04-13 E-Therapeutics Plc Nucleic acids containing abasic nucleosides
CA3259162A1 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc NUCLEIC ACID COMPOUNDS
CA3259147A1 (en) * 2022-07-27 2024-02-01 E-Therapeutics Plc NUCLEIC ACID COMPOUNDS
EP4562147A1 (en) * 2022-07-27 2025-06-04 E-Therapeutics plc Nucleic acid compounds
WO2024165571A2 (en) * 2023-02-06 2024-08-15 E-Therapeutics Plc Inhibitors of expression and/or function
WO2025125576A2 (en) * 2023-12-15 2025-06-19 E-Therapeutics Plc Inhibitors of expression and/or function

Family Cites Families (84)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4475196A (en) 1981-03-06 1984-10-02 Zor Clair G Instrument for locating faults in aircraft passenger reading light and attendant call control system
US4447233A (en) 1981-04-10 1984-05-08 Parker-Hannifin Corporation Medication infusion pump
US4439196A (en) 1982-03-18 1984-03-27 Merck & Co., Inc. Osmotic drug delivery system
US4447224A (en) 1982-09-20 1984-05-08 Infusaid Corporation Variable flow implantable infusion apparatus
US4487603A (en) 1982-11-26 1984-12-11 Cordis Corporation Implantable microinfusion pump system
US4486194A (en) 1983-06-08 1984-12-04 James Ferrara Therapeutic device for administering medicaments through the skin
US4666828A (en) 1984-08-15 1987-05-19 The General Hospital Corporation Test for Huntington's disease
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4801531A (en) 1985-04-17 1989-01-31 Biotechnology Research Partners, Ltd. Apo AI/CIII genomic polymorphisms predictive of atherosclerosis
US4959217A (en) 1986-05-22 1990-09-25 Syntex (U.S.A.) Inc. Delayed/sustained release of macromolecules
US4925678A (en) 1987-04-01 1990-05-15 Ranney David F Endothelial envelopment drug carriers
US5080646A (en) 1988-10-03 1992-01-14 Alza Corporation Membrane for electrotransport transdermal drug delivery
US5272057A (en) 1988-10-14 1993-12-21 Georgetown University Method of detecting a predisposition to cancer by the use of restriction fragment length polymorphism of the gene for human poly (ADP-ribose) polymerase
US5270030A (en) 1988-12-29 1993-12-14 Bio-Technology General Corp. Fibrin binding domain polypeptide and method of producing
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5192659A (en) 1989-08-25 1993-03-09 Genetype Ag Intron sequence analysis method for detection of adjacent and remote locus alleles as haplotypes
US5167616A (en) 1989-12-14 1992-12-01 Alza Corporation Iontophoretic delivery method
US5378825A (en) 1990-07-27 1995-01-03 Isis Pharmaceuticals, Inc. Backbone modified oligonucleotide analogs
US5225182A (en) 1991-10-31 1993-07-06 Sharma Yash P Vectored drug delivery system using a cephaloplastin linking agent and a methed of using the system
KR940703846A (ko) 1991-12-24 1994-12-12 비. 린네 파샬 갭(gap)이 형성된 2′ 변성된 올리고뉴클레오티드(gapped 2′ modifed oligonucleotides)
JPH08500481A (ja) 1992-05-11 1996-01-23 リボザイム・ファーマシューティカルズ・インコーポレーテッド ウイルスの複製を阻害するための方法および薬剤
US6235886B1 (en) 1993-09-03 2001-05-22 Isis Pharmaceuticals, Inc. Methods of synthesis and use
US5593972A (en) 1993-01-26 1997-01-14 The Wistar Institute Genetic immunization
DE69431669T2 (de) 1993-09-02 2003-10-23 Ribozyme Pharmaceuticals, Inc. Enzymatische nukleiksaüre die nicht-nukleotide enthaltet
US5998203A (en) 1996-04-16 1999-12-07 Ribozyme Pharmaceuticals, Inc. Enzymatic nucleic acids containing 5'-and/or 3'-cap structures
US20050032067A1 (en) 2002-11-05 2005-02-10 Prakash Thazha P. Non-phosphorous-linked oligomeric compounds and their use in gene modulation
US5898031A (en) 1996-06-06 1999-04-27 Isis Pharmaceuticals, Inc. Oligoribonucleotides for cleaving RNA
US20050042647A1 (en) 1996-06-06 2005-02-24 Baker Brenda F. Phosphorous-linked oligomeric compounds and their use in gene modulation
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6251666B1 (en) 1997-03-31 2001-06-26 Ribozyme Pharmaceuticals, Inc. Nucleic acid catalysts comprising L-nucleotide analogs
AU9063398A (en) 1997-09-12 1999-04-05 Exiqon A/S Oligonucleotide analogues
US6506559B1 (en) 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
EP2314700A1 (en) 1999-01-28 2011-04-27 Medical College of Georgia Research Institute, Inc Composition and method for in vivo and in vitro attenuation of gene expression using double stranded RNA
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
RU2233844C2 (ru) 1999-02-12 2004-08-10 Санкио Компани Лимитед Новые нуклеозидные и олигонуклеотидные аналоги
WO2000049035A1 (en) 1999-02-19 2000-08-24 The General Hospital Corporation Gene silencing
JP2000290185A (ja) * 1999-04-08 2000-10-17 Sankyo Co Ltd Tggg配列を持つ修飾オリゴデオキシリボヌクレオチドを含有する抗エイズ剤
KR20010112944A (ko) 1999-04-21 2001-12-22 이곤 이 버그 폴리뉴클레오티드 서열의 기능을 억제하기 위한 방법 및조성물
WO2001029058A1 (en) 1999-10-15 2001-04-26 University Of Massachusetts Rna interference pathway genes as tools for targeted genetic interference
GB9925459D0 (en) 1999-10-27 1999-12-29 Plant Bioscience Ltd Gene silencing
ES2319858T3 (es) 1999-11-02 2009-05-14 Novartis Vaccines And Diagnostics, Inc. Receptor cpg (cpg-r) y procedimientos relacionados con el mismo.
GB9927444D0 (en) 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
US8202979B2 (en) 2002-02-20 2012-06-19 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
US20050020525A1 (en) 2002-02-20 2005-01-27 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
JP2003526367A (ja) 2000-03-16 2003-09-09 ジェネティカ インコーポレイテッド Rna干渉の方法とrna干渉組成物
KR20080023768A (ko) 2000-03-30 2008-03-14 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Rna 간섭의 rna 서열 특이적인 매개체
US6693187B1 (en) 2000-10-17 2004-02-17 Lievre Cornu Llc Phosphinoamidite carboxlates and analogs thereof in the synthesis of oligonucleotides having reduced internucleotide charge
KR100909681B1 (ko) 2000-12-01 2009-07-29 막스-플랑크-게젤샤프트 츄어 푀르더룽 데어 비쎈샤프텐 에.파우. Rna 간섭을 매개하는 작은 rna 분자
US20070032441A1 (en) 2001-05-18 2007-02-08 Sirna Therapeutics, Inc. Rna interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (sina)
US20040019001A1 (en) 2002-02-20 2004-01-29 Mcswiggen James A. RNA interference mediated inhibition of protein typrosine phosphatase-1B (PTP-1B) gene expression using short interfering RNA
CN1612930A (zh) 2001-11-21 2005-05-04 三菱化学株式会社 抑制基因表达的方法
WO2003064621A2 (en) 2002-02-01 2003-08-07 Ambion, Inc. HIGH POTENCY siRNAS FOR REDUCING THE EXPRESSION OF TARGET GENES
DK1554381T3 (da) * 2002-02-07 2010-03-29 Sirnasense As Posttranskriptionel silencing af vævsfaktorekspression med korte interfererende RNA'er
US20050096289A1 (en) * 2002-02-07 2005-05-05 Hans Prydz Methods and compositions for modulating tissue factor
EP1432724A4 (en) 2002-02-20 2006-02-01 Sirna Therapeutics Inc INTERFERENCE MEDIATION INHIBITION OF GENE RNA FROM MAP KINASE
US7452987B2 (en) 2002-08-05 2008-11-18 Silence Therapeutics Aktiengesellschaft (Ag) Interfering RNA molecules
US9150605B2 (en) 2002-11-05 2015-10-06 Isis Pharmaceuticals, Inc. Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
EP2213738B1 (en) 2002-11-14 2012-10-10 Dharmacon, Inc. siRNA molecules targeting Bcl-2
US20050233342A1 (en) 2003-03-07 2005-10-20 Muthiah Manoharan Methods of preventing off-target gene silencing
DK1606406T4 (da) 2003-03-21 2013-12-16 Santaris Pharma As Short Interfering RNA (siRNA) Analogues
ATE485394T1 (de) 2003-06-02 2010-11-15 Univ Massachusetts Verfahren und zusammensetzungen zur verbesserung der wirksamkeit und spezifität von fnai
AU2004248136B2 (en) 2003-06-02 2011-09-15 University Of Massachusetts Methods and compositions for controlling efficacy of RNA silencing
US20060241072A1 (en) 2003-06-20 2006-10-26 Isis Pharmaceuticals, Inc. Oligomeric compounds for use in gene modulation
KR101147147B1 (ko) 2004-04-01 2012-05-25 머크 샤프 앤드 돔 코포레이션 Rna 간섭의 오프 타겟 효과 감소를 위한 변형된폴리뉴클레오타이드
CA2569419A1 (en) 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
WO2006020768A2 (en) 2004-08-10 2006-02-23 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides
KR101094617B1 (ko) 2004-08-16 2011-12-15 사일런스 테라퓨틱스 아게 알티피801 억제제의 치료적 용도
US20110313024A1 (en) 2004-08-20 2011-12-22 Leonid Beigelman RNA INTERFERENCE MEDIATED INHIBITION OF PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)
WO2006047842A2 (en) 2004-11-08 2006-05-11 K.U. Leuven Research And Development Modified nucleosides for rna interference
US20090176725A1 (en) * 2005-08-17 2009-07-09 Sirna Therapeutics Inc. Chemically modified short interfering nucleic acid molecules that mediate rna interference
SG165394A1 (en) * 2005-09-16 2010-10-28 Coley Pharm Gmbh Modulation of immunostimulatory properties of short interfering ribonucleic acid (sirna) by nucleotide modification
US20070259827A1 (en) 2006-01-25 2007-11-08 University Of Massachusetts Compositions and methods for enhancing discriminatory RNA interference
GB0608838D0 (en) 2006-05-04 2006-06-14 Novartis Ag Organic compounds
EP2046993A4 (en) 2006-07-07 2010-11-17 Univ Massachusetts RNA SILENCING COMPOSITIONS, AND METHODS OF TREATING HUNTINGTON CHOREA
JP2010507387A (ja) 2006-10-25 2010-03-11 クアーク・ファーマスーティカルス、インコーポレイテッド 新規のsiRNAおよびその使用方法
US20100292301A1 (en) 2007-02-28 2010-11-18 Elena Feinstein Novel sirna structures
US7812002B2 (en) 2007-03-21 2010-10-12 Quark Pharmaceuticals, Inc. Oligoribonucleotide inhibitors of NRF2 and methods of use thereof for treatment of cancer
US8614309B2 (en) 2007-10-03 2013-12-24 Quark Pharmaceuticals, Inc. Double-stranded RNA directed to CASP2 and methods of use thereof
US8188060B2 (en) * 2008-02-11 2012-05-29 Dharmacon, Inc. Duplex oligonucleotides with enhanced functionality in gene regulation
EP2504435B1 (en) 2009-11-26 2019-11-13 Quark Pharmaceuticals, Inc. Sirna compounds comprising terminal substitutions
TWI611021B (zh) 2009-12-09 2018-01-11 日東電工股份有限公司 Hsp47表現之調節
AU2011338682B2 (en) 2010-12-06 2017-04-27 Quark Pharmaceuticals, Inc. Double stranded oligonucleotide compounds comprising threose modifications

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