CN114072129A - 可用于治疗异染性脑白质营养不良的组合物 - Google Patents

可用于治疗异染性脑白质营养不良的组合物 Download PDF

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CN114072129A
CN114072129A CN202080048744.4A CN202080048744A CN114072129A CN 114072129 A CN114072129 A CN 114072129A CN 202080048744 A CN202080048744 A CN 202080048744A CN 114072129 A CN114072129 A CN 114072129A
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vector
seq
raav
certain embodiments
sequence
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J·霍尔多瓦
J·威尔逊
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University of Pennsylvania Penn
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University of Pennsylvania Penn
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CN202080048744.4A 2019-05-03 2020-05-02 可用于治疗异染性脑白质营养不良的组合物 Pending CN114072129A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962843091P 2019-05-03 2019-05-03
US62/843091 2019-05-03
PCT/US2020/031207 WO2020227166A1 (en) 2019-05-03 2020-05-02 Compositions useful in treatment of metachromatic leukodystrophy

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CN114072129A true CN114072129A (zh) 2022-02-18

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US (2) US12338451B2 (https=)
EP (1) EP3962454A4 (https=)
JP (1) JP7715640B2 (https=)
KR (1) KR20220020261A (https=)
CN (1) CN114072129A (https=)
AR (1) AR118850A1 (https=)
AU (1) AU2020270421A1 (https=)
BR (1) BR112021021908A2 (https=)
CA (1) CA3135539A1 (https=)
CL (1) CL2021002881A1 (https=)
CO (1) CO2021016323A2 (https=)
IL (1) IL287715A (https=)
MX (1) MX2021013420A (https=)
PE (1) PE20220930A1 (https=)
SG (1) SG11202111867XA (https=)
TW (1) TW202108760A (https=)
WO (1) WO2020227166A1 (https=)

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KR20230170022A (ko) * 2021-04-12 2023-12-18 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 척수 및 연수 근위축증(sbma) 치료에 유용한 조성물
RU2769577C1 (ru) * 2021-06-01 2022-04-04 федеральное государственное автономное образовательное учреждение высшего образования "Казанский (Приволжский) федеральный университет" (ФГАОУ ВО КФУ) Препарат для лечения метахроматической лейкодистрофии и способ ее лечения
IL308787A (en) * 2021-06-08 2024-01-01 Univ Pennsylvania Recombinant adeno-associated viruses for Leish-Nihan disorders and their uses
US20250177495A1 (en) * 2022-01-10 2025-06-05 The Trustees Of The University Of Pennsylvania Compositions useful in treatment of metachromatic leukodystrophy
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