RU2743398C2 - Генная терапия нейродегенеративных нарушений - Google Patents

Генная терапия нейродегенеративных нарушений Download PDF

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RU2743398C2
RU2743398C2 RU2016140850A RU2016140850A RU2743398C2 RU 2743398 C2 RU2743398 C2 RU 2743398C2 RU 2016140850 A RU2016140850 A RU 2016140850A RU 2016140850 A RU2016140850 A RU 2016140850A RU 2743398 C2 RU2743398 C2 RU 2743398C2
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aav
subject
vector
cells
mice
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RU2016140850A
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RU2016140850A3 (enExample
RU2016140850A (ru
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Марко А. ПАССИНИ
Лэмайа ШИХАБУДДИН
Сэн Х. ЧЭН
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Джензим Корпорейшн
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    • A—HUMAN NECESSITIES
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
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    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14133—Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect

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RU2016140850A 2009-05-02 2010-04-27 Генная терапия нейродегенеративных нарушений RU2743398C2 (ru)

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Application Number Priority Date Filing Date Title
US17498209P 2009-05-02 2009-05-02
US61/174,982 2009-05-02
US26805909P 2009-06-08 2009-06-08
US61/268,059 2009-06-08

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JP6754361B2 (ja) 2014-12-16 2020-09-09 ボード オブ リージェンツ オブ ザ ユニバーシティ オブ ネブラスカ 若年型バッテン病のための遺伝子療法
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JP7327803B2 (ja) 2017-05-09 2023-08-16 ユニバーシティ オブ マサチューセッツ 筋萎縮性側索硬化症（ａｌｓ）を処置する方法
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