EA202192346A1 - DELIVERY OF -SARCOGLICAN USING A VECTOR BASED ON ADENO ASSOCIATED VIRUS AND TREATMENT OF MUSCULAR DYSTROPHY - Google Patents

DELIVERY OF -SARCOGLICAN USING A VECTOR BASED ON ADENO ASSOCIATED VIRUS AND TREATMENT OF MUSCULAR DYSTROPHY

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Publication number
EA202192346A1
EA202192346A1 EA202192346A EA202192346A EA202192346A1 EA 202192346 A1 EA202192346 A1 EA 202192346A1 EA 202192346 A EA202192346 A EA 202192346A EA 202192346 A EA202192346 A EA 202192346A EA 202192346 A1 EA202192346 A1 EA 202192346A1
Authority
EA
Eurasian Patent Office
Prior art keywords
treatment
muscular dystrophy
sarcoglican
delivery
associated virus
Prior art date
Application number
EA202192346A
Other languages
Russian (ru)
Inventor
Луиза Родино-Клапак
Джерри Р. Менделл
Original Assignee
Рисерч Инститьют Эт Нэшнуайд Чилдрен'С Хоспитал
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 Рисерч Инститьют Эт Нэшнуайд Чилдрен'С Хоспитал filed Critical Рисерч Инститьют Эт Нэшнуайд Чилдрен'С Хоспитал
Priority claimed from PCT/US2020/019892 external-priority patent/WO2020176614A1/en
Publication of EA202192346A1 publication Critical patent/EA202192346A1/en

Links

Abstract

Описаны способы лечения мышечной дистрофии, включающие введение вектора рекомбинантного AAV (rAAV) scAAVrh74.MHCK7.hSGCB, способы экспрессии гена бета-саркогликана у пациента, фармацевтические композиции, содержащие rAAV, и способы получения rAAV.Methods for the treatment of muscular dystrophy are described, including the introduction of the recombinant AAV (rAAV) vector scAAVrh74.MHCK7.hSGCB, methods for expressing the beta-sarcoglycan gene in a patient, pharmaceutical compositions containing rAAV, and methods for producing rAAV.

EA202192346A 2019-10-04 2020-02-26 DELIVERY OF -SARCOGLICAN USING A VECTOR BASED ON ADENO ASSOCIATED VIRUS AND TREATMENT OF MUSCULAR DYSTROPHY EA202192346A1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962910779P 2019-10-04 2019-10-04
PCT/US2020/019892 WO2020176614A1 (en) 2019-02-26 2020-02-26 Adeno-associated virus vector delivery of b-sarcoglycan and the treatment of muscular dystrophy

Publications (1)

Publication Number Publication Date
EA202192346A1 true EA202192346A1 (en) 2021-12-07

Family

ID=80631179

Family Applications (1)

Application Number Title Priority Date Filing Date
EA202192346A EA202192346A1 (en) 2019-10-04 2020-02-26 DELIVERY OF -SARCOGLICAN USING A VECTOR BASED ON ADENO ASSOCIATED VIRUS AND TREATMENT OF MUSCULAR DYSTROPHY

Country Status (1)

Country Link
EA (1) EA202192346A1 (en)

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