EA201791805A1 - ВАРИАНТ СРЕДСТВА ДЛЯ RNAi - Google Patents

ВАРИАНТ СРЕДСТВА ДЛЯ RNAi

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Publication number
EA201791805A1
EA201791805A1 EA201791805A EA201791805A EA201791805A1 EA 201791805 A1 EA201791805 A1 EA 201791805A1 EA 201791805 A EA201791805 A EA 201791805A EA 201791805 A EA201791805 A EA 201791805A EA 201791805 A1 EA201791805 A1 EA 201791805A1
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EA
Eurasian Patent Office
Prior art keywords
rnai
sequence
guide
huntington
disease
Prior art date
Application number
EA201791805A
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English (en)
Inventor
Лайза М. Стенек
Адам Палермо
Бренда Ричардс
Серджио Пабло Сарди
Кэтрин О'риордан
Антониус Сонг
Original Assignee
Джензим Корпорейшн
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Publication date
Application filed by Джензим Корпорейшн filed Critical Джензим Корпорейшн
Publication of EA201791805A1 publication Critical patent/EA201791805A1/ru

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Abstract

В данном документе предусматриваются молекулы для RNAi, включающие в себя первую цепь, содержащую направляющую последовательность, и вторую цепь, содержащую последовательность, не являющуюся направляющей, которая содержит выпячивание напротив затравочной области направляющей последовательности; например напротив последовательности расщепления. В некоторых аспектах настоящее изобретение обеспечивает RNAi для лечения болезни Хантингтона. В данном документе предусматриваются кассеты экспрессии, векторы (например, векторы на основе rAAV, рекомбинантного аденовируса, рекомбинантного лентивируса и рекомбинантного HSV), клетки, вирусные частицы и фармацевтические композиции, содержащие RNAi. Кроме того, в данном документе дополнительно предусматриваются способы и наборы, связанные с применением RNAi, например, для лечения болезни Хантингтона.
EA201791805A 2015-02-10 2016-02-09 ВАРИАНТ СРЕДСТВА ДЛЯ RNAi EA201791805A1 (ru)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562114578P 2015-02-10 2015-02-10
PCT/US2016/017207 WO2016130589A2 (en) 2015-02-10 2016-02-09 VARIANT RNAi

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EA201791805A1 true EA201791805A1 (ru) 2018-05-31

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EA201791805A EA201791805A1 (ru) 2015-02-10 2016-02-09 ВАРИАНТ СРЕДСТВА ДЛЯ RNAi

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US (4) US10450563B2 (ru)
EP (1) EP3256588A2 (ru)
JP (2) JP2018506304A (ru)
KR (1) KR102670852B1 (ru)
CN (1) CN107438671B (ru)
AR (1) AR103646A1 (ru)
AU (2) AU2016219396B2 (ru)
BR (1) BR112017017028A2 (ru)
CA (1) CA2976075A1 (ru)
CL (1) CL2017002027A1 (ru)
CO (1) CO2017009083A2 (ru)
CR (1) CR20170406A (ru)
EA (1) EA201791805A1 (ru)
EC (1) ECSP17059343A (ru)
HK (1) HK1247641A1 (ru)
IL (1) IL253893B (ru)
MA (1) MA40819B1 (ru)
MX (2) MX2017010369A (ru)
MY (2) MY194175A (ru)
PE (1) PE20171382A1 (ru)
PH (1) PH12017501432A1 (ru)
SG (3) SG10201907396XA (ru)
TN (1) TN2017000354A1 (ru)
TW (1) TWI781079B (ru)
UY (1) UY36554A (ru)
WO (1) WO2016130589A2 (ru)

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ES2879636T3 (es) 2014-05-02 2021-11-22 Genzyme Corp Vectores AAV para la terapia génica de la retina y el SNC
EP3540063A1 (en) 2014-12-24 2019-09-18 uniQure IP B.V. Rnai induced huntingtin gene suppression
JP2018506304A (ja) 2015-02-10 2018-03-08 ジェンザイム・コーポレーション バリアントＲＮＡｉ
AR113134A1 (es) 2017-09-22 2020-01-29 Genzyme Corp Arni variante

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