WO2002055693A3 - Verfahren zur hemmung der expression eines zielgens - Google Patents

Verfahren zur hemmung der expression eines zielgens Download PDF

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Publication number
WO2002055693A3
WO2002055693A3 PCT/EP2002/000152 EP0200152W WO02055693A3 WO 2002055693 A3 WO2002055693 A3 WO 2002055693A3 EP 0200152 W EP0200152 W EP 0200152W WO 02055693 A3 WO02055693 A3 WO 02055693A3
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WO
WIPO (PCT)
Prior art keywords
target gene
expression
dsrna
inhibiting
dual
Prior art date
Application number
PCT/EP2002/000152
Other languages
English (en)
French (fr)
Other versions
WO2002055693A2 (de
Inventor
Roland Kreutzer
Stephan Limmer
Sylvia Rost
Philipp Hadwiger
Original Assignee
Ribopharma Ag
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=27437921&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=WO2002055693(A3) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Priority claimed from DE10100586A external-priority patent/DE10100586C1/de
Priority to DE10163098A priority Critical patent/DE10163098B4/de
Priority to EP02710786A priority patent/EP1352061B9/de
Priority to AU2002229701A priority patent/AU2002229701B2/en
Priority to JP2002556740A priority patent/JP4210116B2/ja
Application filed by Ribopharma Ag filed Critical Ribopharma Ag
Priority to CA002432350A priority patent/CA2432350A1/en
Priority to DE50206993T priority patent/DE50206993D1/de
Priority to DE10230997A priority patent/DE10230997A1/de
Priority to DE10230996A priority patent/DE10230996A1/de
Publication of WO2002055693A2 publication Critical patent/WO2002055693A2/de
Priority to PCT/EP2002/011432 priority patent/WO2003033700A1/de
Priority to PCT/EP2002/011973 priority patent/WO2003035876A1/de
Priority to CNA028251636A priority patent/CN1604783A/zh
Priority to PCT/EP2002/011972 priority patent/WO2003035083A1/de
Priority to PCT/EP2002/011971 priority patent/WO2003035082A1/de
Priority to US10/493,686 priority patent/US20050119202A1/en
Priority to EP02801917A priority patent/EP1438056A1/de
Priority to CNA028261798A priority patent/CN1608131A/zh
Priority to CNA02826181XA priority patent/CN1608133A/zh
Priority to PCT/EP2002/011968 priority patent/WO2003035868A1/de
Priority to EP02785312A priority patent/EP1438406A1/de
Priority to JP2003538370A priority patent/JP2005506385A/ja
Priority to JP2003538376A priority patent/JP2005506087A/ja
Priority to JP2003537650A priority patent/JP2005512976A/ja
Priority to EP02785313A priority patent/EP1438409A1/de
Priority to US10/493,768 priority patent/US20040248835A1/en
Priority to EP10011813.2A priority patent/EP2264173A3/de
Priority to PCT/EP2002/011970 priority patent/WO2003035870A1/de
Priority to PCT/EP2002/011969 priority patent/WO2003035869A1/de
Priority to EP02779511A priority patent/EP1438405A1/de
Priority to US10/384,512 priority patent/US7348314B2/en
Priority to US10/384,339 priority patent/US7829693B2/en
Priority to US10/384,463 priority patent/US7763590B2/en
Priority to US10/384,434 priority patent/US20040121348A1/en
Publication of WO2002055693A3 publication Critical patent/WO2002055693A3/de
Priority to US10/382,634 priority patent/US20040038921A1/en
Priority to US10/666,458 priority patent/US20040126791A1/en
Priority to US11/747,549 priority patent/US20080070856A1/en
Priority to US11/959,936 priority patent/US7745418B2/en
Priority to US12/631,689 priority patent/US8273868B2/en
Priority to US12/817,009 priority patent/US7994309B2/en
Priority to US12/894,018 priority patent/US8546143B2/en
Priority to US13/177,316 priority patent/US8273870B2/en
Priority to US13/594,150 priority patent/US20130064879A1/en
Priority to US13/594,140 priority patent/US20120322858A1/en
Priority to US14/012,994 priority patent/US9074213B2/en
Priority to US14/737,304 priority patent/US9587240B2/en

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    • C—CHEMISTRY; METALLURGY
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    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
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    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
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    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12N15/1131—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 against viruses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/53—Physical structure partially self-complementary or closed
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

Abstract

Die Erfindung betrifft ein Verfahren zur Hemmung der Expression eines Zielgens in einer Zelle umfassend die folgenden Schritte: Einführen mindestens einer doppelstängigen Ribonukleinsäure (dsRNA I) in einer zur Hemmung der Expression des Zielgens ausreichenden Menge, wobei die dsRNA I eine doppelsträngige aus höchstens 49 aufeinanderfolgenden Nukleotidpaaren gebildete Struktur aufweist, und wobei ein Strang (as1) oder zumindest ein Abschnitt des einen Strangs (as1) der doppelsträngigen Struktur komplementär zum Sinn-Strang des Zielgens ist, und wobei die dsRNA am einen Ende (E1) der dsRNA I einen aus 1 bis 4 Nukeotiden gebildeten überhang aufweist.
PCT/EP2002/000152 1999-11-24 2002-01-09 Verfahren zur hemmung der expression eines zielgens WO2002055693A2 (de)

Priority Applications (44)

Application Number Priority Date Filing Date Title
DE10163098A DE10163098B4 (de) 2001-10-12 2001-12-20 Verfahren zur Hemmung der Replikation von Viren
DE50206993T DE50206993D1 (de) 2001-01-09 2002-01-09 Verfahren zur hemmung der expression eine zielgens
CA002432350A CA2432350A1 (en) 2001-01-09 2002-01-09 Method for inhibiting the expression of a target gene
AU2002229701A AU2002229701B2 (en) 2001-01-09 2002-01-09 Method for inhibiting the expression of a target gene
JP2002556740A JP4210116B2 (ja) 2001-01-09 2002-01-09 標的遺伝子の発現阻害方法
EP02710786A EP1352061B9 (de) 2001-01-09 2002-01-09 Verfahren zur hemmung der expression eine zielgens
DE10230997A DE10230997A1 (de) 2001-10-26 2002-07-09 Medikament zur Erhöhung der Wirksamkeit eines Rezeptor-vermittelt Apoptose in Tumorzellen auslösenden Arzneimittels
DE10230996A DE10230996A1 (de) 2001-10-26 2002-07-09 Medikament zur Behandlung eines Pankreaskarzinoms
PCT/EP2002/011432 WO2003033700A1 (de) 2001-10-12 2002-10-11 Verfahren zur hemmung der replikation von viren
PCT/EP2002/011970 WO2003035870A1 (de) 2001-10-26 2002-10-25 Medikament zur behandlung eines pankreaskarzinoms
EP02779511A EP1438405A1 (de) 2001-01-09 2002-10-25 Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens
EP02785312A EP1438406A1 (de) 2001-10-26 2002-10-25 Medikament zur behandlung eines pankreaskarzinoms
JP2003538376A JP2005506087A (ja) 2001-10-26 2002-10-25 プラス鎖ｒｎａウイルスによる感染症を処置するための２本鎖リボ核酸の使用
PCT/EP2002/011969 WO2003035869A1 (de) 2001-10-26 2002-10-25 Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens
PCT/EP2002/011972 WO2003035083A1 (de) 2001-10-26 2002-10-25 Medikament zur behandlung einer fibrotischen erkrankung durch rna interferenz
PCT/EP2002/011971 WO2003035082A1 (de) 2001-10-26 2002-10-25 Medikament zur hemmung der expression eines zielgens
US10/493,686 US20050119202A1 (en) 2001-10-26 2002-10-25 Medicament to treat a fibrotic disease
EP02801917A EP1438056A1 (de) 2001-10-26 2002-10-25 Medikament zur behandlung einer fibrotischen erkrankung durch rna interferenz
CNA028261798A CN1608131A (zh) 2001-10-26 2002-10-25 治疗胰腺癌的药物
CNA02826181XA CN1608133A (zh) 2001-10-26 2002-10-25 双链核糖核酸用于治疗正(+)链rna病毒感染的用途
PCT/EP2002/011968 WO2003035868A1 (de) 2001-10-26 2002-10-25 Medikament zur erhöhung der wirksamkeit eines rezeptor-vermittelt apoptose in tumorzellen auslösenden arzeimittels
CNA028251636A CN1604783A (zh) 2001-10-26 2002-10-25 通过rna干扰治疗纤维化疾病的药物
JP2003538370A JP2005506385A (ja) 2001-10-26 2002-10-25 膵臓癌を処置するための医薬
PCT/EP2002/011973 WO2003035876A1 (de) 2001-10-26 2002-10-25 Verwendung einer doppelsträngigen ribonukleinsäure zur behandlung einer infektion mit einem (+)-strang-rna-virus
JP2003537650A JP2005512976A (ja) 2001-10-26 2002-10-25 Ｒｎａ干渉により線維化疾患を処置するための医薬
EP02785313A EP1438409A1 (de) 2001-10-26 2002-10-25 Verwendung einer doppelsträngigen ribonukleinsäure zur behandlung einer infektion mit einem (+)-strang-rna-virus
US10/493,768 US20040248835A1 (en) 2001-10-26 2002-10-25 Use of a double-stranded ribonucleic acid for treating an infection with a positivestrand rna-virus
EP10011813.2A EP2264173A3 (de) 2001-01-09 2002-10-25 Verwendung einer doppelsträngigen Ribonukleinsäure zur gezielten Hemmung der Expression eines vorgegebenen Zielgens
US10/384,463 US7763590B2 (en) 2001-10-12 2003-03-07 Compositions and methods for inhibiting expression of a mutant gene
US10/384,512 US7348314B2 (en) 2001-10-12 2003-03-07 Compositions and methods for inhibiting viral replication
US10/384,434 US20040121348A1 (en) 2001-10-26 2003-03-07 Compositions and methods for treating pancreatic cancer
US10/384,339 US7829693B2 (en) 1999-11-24 2003-03-07 Compositions and methods for inhibiting expression of a target gene
US10/382,634 US20040038921A1 (en) 2001-10-26 2003-08-11 Composition and method for inhibiting expression of a target gene
US10/666,458 US20040126791A1 (en) 2001-10-26 2003-09-19 Compositions and methods for treating trail-resistant cancer cells
US11/747,549 US20080070856A1 (en) 2001-10-26 2007-05-11 Medicament to treat a fibrotic disease
US11/959,936 US7745418B2 (en) 2001-10-12 2007-12-19 Compositions and methods for inhibiting viral replication
US12/631,689 US8273868B2 (en) 2001-10-12 2009-12-04 Compositions and methods for inhibiting viral replication
US12/817,009 US7994309B2 (en) 2001-10-26 2010-06-16 Compositions and methods for inhibiting expression of a mutant gene
US12/894,018 US8546143B2 (en) 2001-01-09 2010-09-29 Compositions and methods for inhibiting expression of a target gene
US13/177,316 US8273870B2 (en) 2001-10-26 2011-07-06 Compositions and methods for inhibiting expression of a mutant gene
US13/594,140 US20120322858A1 (en) 2001-10-26 2012-08-24 Compositions and Methods for Inhibiting Expression of a Mutant Gene
US13/594,150 US20130064879A1 (en) 2001-10-12 2012-08-24 Compositions and Methods for Inhibiting Viral Replication
US14/012,994 US9074213B2 (en) 2001-01-09 2013-08-28 Compositions and methods for inhibiting expression of a target gene
US14/737,304 US9587240B2 (en) 2001-01-09 2015-06-11 Compositions and methods for inhibiting expression of a target gene

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
DE10100586.5 2001-01-09
DE10100586A DE10100586C1 (de) 2001-01-09 2001-01-09 Verfahren zur Hemmung der Expression eines Ziegens
DE10155280.7 2001-10-26
DE10155280 2001-10-26
DE10158411 2001-11-29
DE10158411.3 2001-11-29
DE10160151A DE10160151A1 (de) 2001-01-09 2001-12-07 Verfahren zur Hemmung der Expression eines vorgegebenen Zielgens
DE10160151.4 2001-12-07

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2002/000151 Continuation WO2002055692A2 (de) 2001-01-09 2002-01-09 Verfahren zur hemmung der expression eines zielgens und medikament zur therapie einer tumorerkrankung

Related Child Applications (2)

Application Number Title Priority Date Filing Date
PCT/EP2002/011969 Continuation WO2003035869A1 (de) 2001-01-09 2002-10-25 Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens
US10/384,339 Continuation-In-Part US7829693B2 (en) 1999-11-24 2003-03-07 Compositions and methods for inhibiting expression of a target gene

Publications (2)

Publication Number Publication Date
WO2002055693A2 WO2002055693A2 (de) 2002-07-18
WO2002055693A3 true WO2002055693A3 (de) 2003-07-17

Family

ID=27437921

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/EP2002/000152 WO2002055693A2 (de) 1999-11-24 2002-01-09 Verfahren zur hemmung der expression eines zielgens

Country Status (8)

Country Link
EP (1) EP1352061B9 (de)
JP (1) JP4210116B2 (de)
CN (1) CN1630724A (de)
AT (1) ATE328075T1 (de)
AU (1) AU2002229701B2 (de)
CA (1) CA2432350A1 (de)
DE (2) DE10160151A1 (de)
WO (1) WO2002055693A2 (de)

Cited By (6)

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US8846894B2 (en) 2002-02-20 2014-09-30 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US8853384B2 (en) 2000-12-01 2014-10-07 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. RNA interference mediating small RNA molecules
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US9273107B2 (en) 2012-12-27 2016-03-01 Ngm Biopharmaceuticals, Inc. Uses and methods for modulating bile acid homeostasis and treatment of bile acid disorders and diseases
US9290557B2 (en) 2012-11-28 2016-03-22 Ngm Biopharmaceuticals, Inc. Compositions comprising variants and fusions of FGF19 polypeptides
US9580483B2 (en) 2011-07-01 2017-02-28 Ngm Biopharmaceuticals, Inc. Methods of using compositions comprising variants and fusions of FGF19 polypeptides for treatment of diabetes

Families Citing this family (168)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
US7601494B2 (en) 1999-03-17 2009-10-13 The University Of North Carolina At Chapel Hill Method of screening candidate compounds for susceptibility to biliary excretion
US7829693B2 (en) 1999-11-24 2010-11-09 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
DE10100586C1 (de) 2001-01-09 2002-04-11 Ribopharma Ag Verfahren zur Hemmung der Expression eines Ziegens
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
ATE450621T2 (de) 2000-03-30 2009-12-15 Whitehead Biomedical Inst Mediatoren von rns-interferenz, die rns- sequenzspezifisch sind
WO2003035869A1 (de) * 2001-10-26 2003-05-01 Ribopharma Ag Verwendung einer doppelsträngigen ribonukleinsäure zur gezielten hemmung der expression eines vorgegebenen zielgens
US7767802B2 (en) 2001-01-09 2010-08-03 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of anti-apoptotic genes
EP2264173A3 (de) * 2001-01-09 2015-01-14 Alnylam Europe AG Verwendung einer doppelsträngigen Ribonukleinsäure zur gezielten Hemmung der Expression eines vorgegebenen Zielgens
US8546143B2 (en) 2001-01-09 2013-10-01 Alnylam Pharmaceuticals, Inc. Compositions and methods for inhibiting expression of a target gene
US20050148530A1 (en) 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US9994853B2 (en) 2001-05-18 2018-06-12 Sirna Therapeutics, Inc. Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
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DE10160151A1 (de) 2003-06-26
EP1352061A2 (de) 2003-10-15
WO2002055693A2 (de) 2002-07-18
CN1630724A (zh) 2005-06-22
ATE328075T1 (de) 2006-06-15
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