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RU2016123086A - Способы и композиции для лечения болезни хантингтона - Google Patents

Способы и композиции для лечения болезни хантингтона Download PDF

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RU2016123086A
RU2016123086A RU2016123086A RU2016123086A RU2016123086A RU 2016123086 A RU2016123086 A RU 2016123086A RU 2016123086 A RU2016123086 A RU 2016123086A RU 2016123086 A RU2016123086 A RU 2016123086A RU 2016123086 A RU2016123086 A RU 2016123086A
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Х. Стив ЧЖАН
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Сангамо Байосайенсиз, Инк.
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Claims (13)

1. Способ увеличения уровней PDE10a, DARPP-32, DRD1 и/или DRD2 в срединных шипиковых нейронах (MSN) субъекта, по меньшей мере, на 30% относительно контроля, при этом способ включает введение субъекту генетического репрессора гена, ассоциированного с болезнью Хантингтона.
2. Способ по п. 1, где уровни PDE10a, DARPP-32, DRD1 и/или DRD2 увеличиваются по меньшей мере на 40% или 50% или более относительно контроля.
3. Способ по п. 1 или 2, где генетический репрессор представляет собой малую молекулу, нуклеиновую кислоту или белок, которые ингибируют нуклеиновую кислоту, кодирующую белок хантингтин (например, генетическую ДНК или мРНК).
4. Способ по любому из пп. 1-3, где генетический репрессор специфически связывается с нуклеиновой кислотой, кодирующей белок хантингтин.
5. Способ по любому из пп. 1-4, где генетический репрессор содержит сконструированный ДНК-связывающий домен.
6. Способ по п. 5, где ДНК-связывающий домен содержит сконструированный белок ”цинковые пальцы”, систему CRISPR/Cas или домен TAL эффектора.
7. Способ по п. 5 или 6, где ДНК-связывающий домен слит с функциональным доменом.
8. Способ по п. 7, где функциональный домен представляет собой домен транскрипционного репрессора или нуклеазу.
9. Способ по любому из пп. 1-8, где генетический репрессор вводят в центральную нервную систему (ЦНС) субъекта.
10. Способ по п. 9, где генетический репрессор вводят в полосатое тело.
11. Способ по любому из пп. 1-10, где генетический репрессор вводят с использованием вирусного или невирусного вектора.
12. Способ по п. 11, где невирусный вектор представляет собой аденоассоциированный вирусный (AAV) вектор.
13. Способ лечения болезни Хантингтона у субъекта, причем способ включает увеличение уровней PDE10a, DARPP-32, DRD1 и/или DRD2 в срединных шипиковых нейронах (MSN) согласно способам по любому из пп. 1-12.
RU2016123086A 2013-11-11 2014-11-11 Способы и композиции для лечения болезни хантингтона RU2693891C1 (ru)

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US201361902704P 2013-11-11 2013-11-11
US61/902,704 2013-11-11
PCT/US2014/064987 WO2015070212A1 (en) 2013-11-11 2014-11-11 Methods and compositions for treating huntington's disease

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US (1) US10369201B2 (ru)
EP (1) EP3068905A4 (ru)
JP (2) JP2016537341A (ru)
KR (1) KR102431079B1 (ru)
CN (1) CN105934524A (ru)
AU (1) AU2014346424B2 (ru)
BR (1) BR112016010175A2 (ru)
CA (1) CA2929179A1 (ru)
MX (1) MX2016006106A (ru)
NZ (1) NZ719477A (ru)
RU (1) RU2693891C1 (ru)
WO (1) WO2015070212A1 (ru)

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