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 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
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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US20090010894A1 (en) * 2005-12-23 2009-01-08 The Parkinson's Institute Methods and systems for identifying compounds that modulate alpha-synuclein aggregation
EP1996612A4 (de) * 2006-03-03 2010-10-20 Univ Kingston Zusammensetzungen zur behandlung von krebs
GB0610183D0 (en) * 2006-05-23 2006-06-28 Isis Innovation Treatment of neurodegenerative diseases
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
MX355683B (es) * 2008-04-18 2018-04-26 Angiochem Inc Composiciones farmacéuticas de paclitaxel, análogos de paclitaxel o conjugados de paclitaxel, y métodos relacionados de preparación y uso.
MX2011004019A (es) 2008-10-15 2011-06-24 Angiochem Inc Conjugados de etoposido y doxorubicina para entrega de farmacos.
EP2346906A4 (de) 2008-10-15 2013-04-24 Angiochem Inc Konjugate aus glp-1-agonisten und ihre verwendung
BRPI0922689A2 (pt) * 2008-12-05 2018-11-06 Angiochem Inc. conjugados de neurotensina ou análogos de neurotensina e usos dos mesmos
CN102300987A (zh) 2008-12-17 2011-12-28 安吉奥开米公司 膜型-1基质金属蛋白抑制剂及其用途
MX2011011023A (es) 2009-04-20 2012-01-20 Angiochem Inc Tratamiento de cancer de ovarios usando un agente anti-cancer conjugado con un analogo de angiopep-2.
US9255266B2 (en) 2009-05-06 2016-02-09 Rutgers, The State University 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
EP2448965A4 (de) 2009-07-02 2015-02-11 Angiochem Inc Multimerpeptid-konjugate und deren verwendungen
EP2486061A4 (de) * 2009-10-06 2013-08-28 Angiochem Inc Zusammensetzungen und verfahren für den transport von therapeutika
EP2380595A1 (de) 2010-04-19 2011-10-26 Nlife Therapeutics S.L. Zusammensetzungen und Verfahren zur selektiven Abgabe von Oligonukleotidmolekülen an spezifische Neuronentypen
AU2011329777B2 (en) 2010-11-17 2016-06-09 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
AU2012340567B2 (en) 2011-11-22 2017-11-23 The Children's Hospital Of Philadelphia Virus vectors for highly efficient transgene delivery
SI3024498T1 (sl) 2013-07-22 2020-07-31 The Children's Hospital Of Philadelphia Različica AAV in sestavki, postopki in uporabe za prenos genov v celice, organe in tkiva
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
IL309741A (en) 2015-06-23 2024-02-01 Childrens Hospital Philadelphia Modified factor IX, and preparations, methods and uses for gene transfer to cells, organs and tissues
WO2018090054A1 (en) 2016-11-14 2018-05-17 Berg Llc Methods for treating parkinson's disease
CA3078371A1 (en) 2017-10-03 2019-04-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
AU2018346105C1 (en) 2017-10-03 2023-10-12 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
EP3692151A4 (de) 2017-10-03 2021-07-14 Prevail Therapeutics, Inc. Gentherapien für lyosomale erkrankungen
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
AU2019208006A1 (en) 2018-01-12 2020-07-23 Bristol-Myers Squibb Company Antisense oligonucleotides targeting alpha-synuclein and uses thereof
MX2020006973A (es) 2018-01-12 2020-09-09 Roche Innovation Ct Copenhagen As Oligonucleotidos antisentido para alfa-sinucleina y usos de los mismos.
NL2022538B1 (en) * 2019-02-08 2020-08-19 Academisch Ziekenhuis Groningen Methods for providing purified viral particles of Semliki Forest Virus (SFV), preparations obtainable thereby, and uses thereof.
AU2020270984A1 (en) 2019-04-10 2021-12-02 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
WO2022268835A1 (en) * 2021-06-21 2022-12-29 Uniqure Biopharma B.V. Gene constructs for silencing alpha-synuclein and uses thereof
EP4342988A1 (de) 2022-09-20 2024-03-27 Eberhard Karls Universität Tübingen, Medizinische Fakultät Zusammensetzungen und verfahren zur modifikation der expression eines snca-gens

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US7605249B2 (en) * 2002-11-26 2009-10-20 Medtronic, Inc. Treatment of neurodegenerative disease through intracranial delivery of siRNA
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Also Published As

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

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