WO2005045034A2 - Traitement medie par interference arn de la maladie de parkinson au moyen d'un petit acide nucleique interferent (sina) - Google Patents

Traitement medie par interference arn de la maladie de parkinson au moyen d'un petit acide nucleique interferent (sina) Download PDF

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Publication number
WO2005045034A2
WO2005045034A2 PCT/US2004/017630 US2004017630W WO2005045034A2 WO 2005045034 A2 WO2005045034 A2 WO 2005045034A2 US 2004017630 W US2004017630 W US 2004017630W WO 2005045034 A2 WO2005045034 A2 WO 2005045034A2
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WIPO (PCT)
Prior art keywords
sina
nucleotides
sina molecule
molecule
rna
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PCT/US2004/017630
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English (en)
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WO2005045034A3 (fr
Inventor
James Mcswiggen
Peter Haeberli
Leonid Beigelman
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Sirna Therapeutics, Inc.
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority claimed from US10/693,059 external-priority patent/US20080039414A1/en
Priority claimed from US10/698,311 external-priority patent/US20040219671A1/en
Priority claimed from US10/720,448 external-priority patent/US8273866B2/en
Priority claimed from US10/727,780 external-priority patent/US20050233329A1/en
Priority claimed from US10/757,803 external-priority patent/US20050020525A1/en
Priority claimed from US10/780,447 external-priority patent/US7491805B2/en
Priority claimed from US10/826,966 external-priority patent/US20050032733A1/en
Priority claimed from PCT/US2004/013456 external-priority patent/WO2005041859A2/fr
Priority claimed from PCT/US2004/016390 external-priority patent/WO2005019453A2/fr
Priority to EP04754277A priority Critical patent/EP1675948A2/fr
Application filed by Sirna Therapeutics, Inc. filed Critical Sirna Therapeutics, Inc.
Publication of WO2005045034A2 publication Critical patent/WO2005045034A2/fr
Publication of WO2005045034A3 publication Critical patent/WO2005045034A3/fr

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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K49/00—Preparations for testing in vivo
    • A61K49/0004—Screening or testing of compounds for diagnosis of disorders, assessment of conditions, e.g. renal clearance, gastric emptying, testing for diabetes, allergy, rheuma, pancreas functions
    • A61K49/0008—Screening agents using (non-human) animal models or transgenic animal models or chimeric hosts, e.g. Alzheimer disease animal model, transgenic model for heart failure
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/332—Abasic residue

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  • Health & Medical Sciences (AREA)
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  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
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  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
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Abstract

La présente invention concerne des méthodes et des réactifs utiles dans la modulation des gènes de la maladie de Parkinson, et notamment de l'expression génique de PARK1 (SNCA), PARK2, PARK7 et/ou PARK5, dans une pluralité d'applications, telles que des applications thérapeutiques et diagnostiques, la validation de cibles et la recherche génomique. Plus particulièrement, l'invention concerne des petites molécules d'acide nucléique, telles que des molécules d'un petit acide nucléique interférent (siNA), d'un petit ARN interférent (siRNA), d'un ARN bicaténaire (dsRNA), d'un micro-ARN (miRNA) et d'un petit ARN en épingle à cheveux (shRNA), permettant de médier l'interférence ARN (RNAi) contre l'expression et/ou l'activité génique de SNCA. Ces petites molécules d'acide nucléique sont utiles dans le diagnostic et le traitement de la maladie de Parkinson (PD) et de n'importe quelle autre maladie ou affection sensible à la modulation de l'expression ou de l'activité de PARK1 (SNCA), PARK2, PARK7 et/ou PARK5.
PCT/US2004/017630 2003-10-23 2004-06-03 Traitement medie par interference arn de la maladie de parkinson au moyen d'un petit acide nucleique interferent (sina) WO2005045034A2 (fr)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP04754277A EP1675948A2 (fr) 2003-10-23 2004-06-03 Traitement medie par interference arn de la maladie de parkinson au moyen d'un petit acide nucleique interferent (sina)

Applications Claiming Priority (20)

Application Number Priority Date Filing Date Title
US10/693,059 2003-10-23
US10/693,059 US20080039414A1 (en) 2002-02-20 2003-10-23 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US10/698,311 2003-10-31
US10/698,311 US20040219671A1 (en) 2002-02-20 2003-10-31 RNA interference mediated treatment of parkinson disease using short interfering nucleic acid (siNA)
US10/720,448 2003-11-24
US10/720,448 US8273866B2 (en) 2002-02-20 2003-11-24 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
US10/727,780 US20050233329A1 (en) 2002-02-20 2003-12-03 Inhibition of gene expression using duplex forming oligonucleotides
US10/727,780 2003-12-03
US10/757,803 US20050020525A1 (en) 2002-02-20 2004-01-14 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US10/757,803 2004-01-14
US54348004P 2004-02-10 2004-02-10
US60/543,480 2004-02-10
US10/780,447 2004-02-13
US10/780,447 US7491805B2 (en) 2001-05-18 2004-02-13 Conjugates and compositions for cellular delivery
US10/826,966 2004-04-16
US10/826,966 US20050032733A1 (en) 2001-05-18 2004-04-16 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA)
PCT/US2004/013456 WO2005041859A2 (fr) 2003-04-30 2004-04-30 Conjugues et compositions
USPCT/US04/13456 2004-04-30
USPCT/US04/16390 2004-05-24
PCT/US2004/016390 WO2005019453A2 (fr) 2001-05-18 2004-05-24 Interference arn a mediation assuree par l'inhibition de genes au moyen de petit acide nucleique interferent (ansi) modifie chimiquement

Publications (2)

Publication Number Publication Date
WO2005045034A2 true WO2005045034A2 (fr) 2005-05-19
WO2005045034A3 WO2005045034A3 (fr) 2005-08-11

Family

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Family Applications (1)

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PCT/US2004/017630 WO2005045034A2 (fr) 2003-10-23 2004-06-03 Traitement medie par interference arn de la maladie de parkinson au moyen d'un petit acide nucleique interferent (sina)

Country Status (2)

Country Link
EP (1) EP1675948A2 (fr)
WO (1) WO2005045034A2 (fr)

Cited By (73)

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WO2005116212A2 (fr) * 2004-05-25 2005-12-08 Medtronic, Inc. Traitement de maladies neurodegeneratives par l'administration intracranienne d'arnic
EP1635763A2 (fr) * 2003-06-09 2006-03-22 Alnylam Pharmaceuticals Inc. Procede de traitement de maladie neurodegenerative
WO2006008124A3 (fr) * 2004-07-19 2006-05-18 Oryzon Genomics Sa Technique diagnostique moleculaire et traitement de la demence a corps de lewy
WO2007119237A1 (fr) * 2006-04-13 2007-10-25 Ramot At Tel Aviv University Ltd. Méthodes et trousses pour diagnostiquer des maladies associées à un stress oxydatif
WO2008109509A1 (fr) * 2007-03-02 2008-09-12 Mdrna, Inc. Composés d'acide nucléique pour inhiber l'expression du gène snca et utilisations de ceux-ci
US7498316B2 (en) 2004-04-06 2009-03-03 University Of Massachusetts Methods and compositions for treating gain-of-function disorders using RNA interference
WO2011019423A2 (fr) 2009-05-20 2011-02-17 Schering Corporation Modulation de récepteurs pilr pour traiter les infections microbiennes
WO2011031600A1 (fr) 2009-09-10 2011-03-17 Schering Corporation Utilisation d'antagonistes de l'il-33 à des fins de traitement des maladies fibrotiques
US7947658B2 (en) 2003-09-12 2011-05-24 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
WO2011084357A1 (fr) 2009-12-17 2011-07-14 Schering Corporation Modulation de pilr pour le traitement de troubles immunitaires
US8058251B2 (en) 2002-11-26 2011-11-15 Kaemmerer William F Devices, systems and methods for improving memory and/or cognitive function through brain delivery of siRNA
US8153606B2 (en) 2008-10-03 2012-04-10 Opko Curna, Llc Treatment of apolipoprotein-A1 related diseases by inhibition of natural antisense transcript to apolipoprotein-A1
US8288354B2 (en) 2005-12-28 2012-10-16 The Scripps Research Institute Natural antisense and non-coding RNA transcripts as drug targets
CN103149368A (zh) * 2005-10-21 2013-06-12 株式会社芳珂 特应性皮炎标记物及其利用技术
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
US8791085B2 (en) 2009-05-28 2014-07-29 Curna, Inc. Treatment of antiviral gene related diseases by inhibition of natural antisense transcript to an antiviral gene
US8791087B2 (en) 2009-08-21 2014-07-29 Curna, Inc. Treatment of ‘C terminus of HSP70-interacting protein’ (CHIP)related diseases by inhibition of natural antisense transcript to CHIP
US8859515B2 (en) 2009-06-24 2014-10-14 Curna, Inc. Treatment of tumor necrosis factor receptor 2 (TNFR2) related diseases by inhibition of natural antisense transcript to TNFR2
US8895528B2 (en) 2010-05-26 2014-11-25 Curna, Inc. Treatment of atonal homolog 1 (ATOH1) related diseases by inhibition of natural antisense transcript to ATOH1
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US8921330B2 (en) 2009-06-26 2014-12-30 Curna, Inc. Treatment of down syndrome gene related diseases by inhibition of natural antisense transcript to a down syndrome gene
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US9044493B2 (en) 2009-08-11 2015-06-02 Curna, Inc. Treatment of Adiponectin related diseases by inhibition of natural antisense transcript to an Adiponectin
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