EA201690842A1 - Псевдотипированный вектор на основе аденоассоциированного вируса aav-5 для генной терапии неврологических заболеваний - Google Patents

Псевдотипированный вектор на основе аденоассоциированного вируса aav-5 для генной терапии неврологических заболеваний

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EA201690842A1
EA201690842A1 EA201690842A EA201690842A EA201690842A1 EA 201690842 A1 EA201690842 A1 EA 201690842A1 EA 201690842 A EA201690842 A EA 201690842A EA 201690842 A EA201690842 A EA 201690842A EA 201690842 A1 EA201690842 A1 EA 201690842A1
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Eurasian Patent Office
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adeno
virus
gene therapy
aav
injection
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EA201690842A
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English (en)
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EA036394B1 (ru
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Бас Блитс
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ЮНИКЬЮРЕ АйПи Б.В.
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Publication of EA201690842A1 publication Critical patent/EA201690842A1/ru
Publication of EA036394B1 publication Critical patent/EA036394B1/ru

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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • 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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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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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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Abstract

Настоящее изобретение относится к способам лечения нарушений, затрагивающих двигательную функцию, как например, двигательную функцию, нарушенную вследствие болезни, травмы головного и/или спинного мозга, с применением генотерапевтического вектора на основе аденоассоциированного вируса (AAV), содержащего капсидные белки аденоассоциированного вируса серотипа 5 (AAV5) и продукт гена, представляющий интерес, фланкированный инвертированными концевыми повторами ITR AAV. В частности, генотерапевтический вектор по настоящему изобретению вводится путем инъекции в цереброспинальную жидкость (ЦСЖ), предпочтительно путем люмбальной инъекции и/или инъекции в мостомозжечковую цистерну cisterna magna.
EA201690842A 2013-10-24 2014-10-24 Псевдотипированный вектор на основе аденоассоциированного вируса aav-5 для генной терапии неврологических заболеваний EA036394B1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP13190141 2013-10-24
PCT/NL2014/050737 WO2015060722A1 (en) 2013-10-24 2014-10-24 Aav-5 pseudotyped vector for gene therapy for neurological diseases

Publications (2)

Publication Number Publication Date
EA201690842A1 true EA201690842A1 (ru) 2016-10-31
EA036394B1 EA036394B1 (ru) 2020-11-05

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EA201690842A EA036394B1 (ru) 2013-10-24 2014-10-24 Псевдотипированный вектор на основе аденоассоциированного вируса aav-5 для генной терапии неврологических заболеваний

Country Status (8)

Country Link
US (1) US20160243260A1 (ru)
EP (1) EP3060669A1 (ru)
JP (2) JP6873699B2 (ru)
CN (1) CN105745326A (ru)
AU (1) AU2014337783B2 (ru)
CA (1) CA2927366A1 (ru)
EA (1) EA036394B1 (ru)
WO (1) WO2015060722A1 (ru)

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US20160243260A1 (en) 2016-08-25
CN105745326A (zh) 2016-07-06
JP2016535587A (ja) 2016-11-17
JP2019216724A (ja) 2019-12-26
JP6873699B2 (ja) 2021-05-19
AU2014337783B2 (en) 2020-07-02
EA036394B1 (ru) 2020-11-05
AU2014337783A1 (en) 2016-05-05
EP3060669A1 (en) 2016-08-31
WO2015060722A1 (en) 2015-04-30

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