ATE439440T1 - Sirna-vermitteltes gen-silencing von alpha- synuklein - Google Patents

Sirna-vermitteltes gen-silencing von alpha- synuklein

Info

Publication number
ATE439440T1
ATE439440T1 AT05816006T AT05816006T ATE439440T1 AT E439440 T1 ATE439440 T1 AT E439440T1 AT 05816006 T AT05816006 T AT 05816006T AT 05816006 T AT05816006 T AT 05816006T AT E439440 T1 ATE439440 T1 AT E439440T1
Authority
AT
Austria
Prior art keywords
synuklein
sirna
alpha
gene silencing
mediated gene
Prior art date
Application number
AT05816006T
Other languages
English (en)
Inventor
Martha Bohn
Mohan Sapru
Original Assignee
Childrens Memorial Hospital
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 Childrens Memorial Hospital filed Critical Childrens Memorial Hospital
Application granted granted Critical
Publication of ATE439440T1 publication Critical patent/ATE439440T1/de

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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • 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
    • C12N2310/11—Antisense
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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 nucleic acids [NA]
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Veterinary Medicine (AREA)
  • Microbiology (AREA)
  • Medicinal Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
AT05816006T 2004-09-29 2005-09-27 Sirna-vermitteltes gen-silencing von alpha- synuklein ATE439440T1 (de)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US61411204P 2004-09-29 2004-09-29
PCT/US2005/034516 WO2006039253A2 (en) 2004-09-29 2005-09-27 Sirna-mediated gene silencing of alpha synuclein

Publications (1)

Publication Number Publication Date
ATE439440T1 true ATE439440T1 (de) 2009-08-15

Family

ID=36142994

Family Applications (1)

Application Number Title Priority Date Filing Date
AT05816006T ATE439440T1 (de) 2004-09-29 2005-09-27 Sirna-vermitteltes gen-silencing von alpha- synuklein

Country Status (6)

Country Link
US (2) US20070172462A1 (de)
EP (1) EP1799826B1 (de)
AT (1) ATE439440T1 (de)
CA (1) CA2580189C (de)
DE (1) DE602005015994D1 (de)
WO (1) WO2006039253A2 (de)

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WO2008033285A2 (en) * 2006-09-15 2008-03-20 The Trustees Of Culumbia University In The City Of New York Delivery of double-stranded rna into the central nervous system
WO2008109509A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting snca gene expression and uses thereof
US9365634B2 (en) * 2007-05-29 2016-06-14 Angiochem Inc. Aprotinin-like polypeptides for delivering agents conjugated thereto to tissues
GB0715809D0 (en) * 2007-08-14 2007-09-26 Univ Leuven Kath Alpha synuclein toxicity
TR201905480T4 (tr) * 2008-04-18 2019-05-21 Angiochem Inc Paklitaksel, paklitaksel analogları veya paklitaksel konjugatlarının farmasötik bileşimleri ve ilgili preparasyon ve kullanım yöntemleri.
BRPI0920209A2 (pt) 2008-10-15 2015-12-22 Angiochem Inc conjugados de agonistas de glp-1 e usos dos mesmos
MX2011004019A (es) 2008-10-15 2011-06-24 Angiochem Inc Conjugados de etoposido y doxorubicina para entrega de farmacos.
CA2745524C (en) * 2008-12-05 2020-06-09 Angiochem Inc. Conjugates of neurotensin or neurotensin analogs and uses thereof
WO2010069074A1 (en) 2008-12-17 2010-06-24 Universite Du Quebec A Montreal Membrane type-1 matrix metalloprotein inhibitors and uses thereof
WO2010121379A1 (en) 2009-04-20 2010-10-28 Angiochem Inc Treatment of ovarian cancer using an anticancer agent conjugated to an angiopep-2 analog
WO2010129791A1 (en) * 2009-05-06 2010-11-11 University Of Medicine And Dentistry Of New Jersey Rna targeting in alpha-synucleinopathies
IT1398977B1 (it) 2009-06-25 2013-03-28 Icgeb Agente anticancro specifico per i tumori cerebrali con meccanismo di soppressione di recq1
AU2010268726A1 (en) 2009-07-02 2012-01-19 Angiochem Inc. Multimeric peptide conjugates and uses thereof
CN102781965A (zh) * 2009-10-06 2012-11-14 安吉奥开米公司 用于转运治疗剂的组合物和方法
EP2380595A1 (de) 2010-04-19 2011-10-26 Nlife Therapeutics S.L. Zusammensetzungen und Verfahren zur selektiven Abgabe von Oligonukleotidmolekülen an spezifische Neuronentypen
CA3077910A1 (en) 2010-11-17 2012-05-24 Ionis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
US20150037257A1 (en) * 2011-06-16 2015-02-05 Sally Mak Compositions and methods for high-throughput screening in skin fibroblasts with an alpha-synuclein triplication
JP6348064B2 (ja) 2011-11-22 2018-06-27 ザ チルドレンズ ホスピタル オブ フィラデルフィア 効率の高いトランスジーン送達のためのウイルスベクター
KR20240090694A (ko) 2013-07-22 2024-06-21 더 칠드런스 호스피탈 오브 필라델피아 변종 ａａｖ 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
EP3091087A1 (de) * 2015-05-08 2016-11-09 Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol Verfahren zur in-vitro-diagnose von synukleinopathien mit alpha-synuklein-gentranskripten
DK3307326T3 (da) 2015-06-15 2020-10-19 Angiochem Inc Fremgangsmåder til behandling af leptomeningeal karcinomatose
WO2016210170A1 (en) 2015-06-23 2016-12-29 The Children's Hospital Of Philadelphia Modified factor ix, and compositions, methods and uses for gene transfer to cells, organs and tissues
EP3538095A4 (de) 2016-11-14 2020-06-17 Berg LLC Verfahren zur behandlung von morbus parkinson
BR112020006671A2 (pt) 2017-10-03 2020-12-01 Prevail Therapeutics, Inc. terapias genéticas para distúrbios lisossômicos
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KR102709597B1 (ko) 2017-10-03 2024-09-26 프리베일 테라퓨틱스, 인크. 리소좀 장애를 위한 유전자 요법
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
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EP4359525A1 (de) * 2021-06-21 2024-05-01 uniQure biopharma B.V. Genkonstrukte zur ausschaltung von alpha-synuklein und verwendungen davon
EP4342988A1 (de) 2022-09-20 2024-03-27 Eberhard Karls Universität Tübingen, Medizinische Fakultät Zusammensetzungen und verfahren zur modifikation der expression eines snca-gens
TW202523337A (zh) * 2023-10-20 2025-06-16 美商星火治療公司 靶向α突觸核蛋白表現之抑制性核酸

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DE60323340D1 (de) * 2002-02-14 2008-10-16 Hope City Verfahren zur herstellung interferierender rna-moleküle in säugetierzellen und therapeutische anwendungen solcher moleküle
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Also Published As

Publication number Publication date
US20070172462A1 (en) 2007-07-26
US20100286242A1 (en) 2010-11-11
EP1799826B1 (de) 2009-08-12
CA2580189C (en) 2013-05-21
DE602005015994D1 (de) 2009-09-24
CA2580189A1 (en) 2006-04-13
US8389487B2 (en) 2013-03-05
WO2006039253A2 (en) 2006-04-13
WO2006039253A3 (en) 2006-11-23
EP1799826A2 (de) 2007-06-27

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