CN114555813A - 治疗雷特综合征的胆固醇24-水解酶的表达载体 - Google Patents

治疗雷特综合征的胆固醇24-水解酶的表达载体 Download PDF

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CN114555813A
CN114555813A CN202080051431.4A CN202080051431A CN114555813A CN 114555813 A CN114555813 A CN 114555813A CN 202080051431 A CN202080051431 A CN 202080051431A CN 114555813 A CN114555813 A CN 114555813A
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vector
treatment
rtt
cholesterol
rett syndrome
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CN202080051431.4A
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Chinese (zh)
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F·皮盖
N·卡蒂埃-拉卡夫
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Centre National de la Recherche Scientifique CNRS
Institut National de la Sante et de la Recherche Medicale INSERM
Sorbonne Universite
Institut du Cerveau et de La Moelle Epiniere ICM
Original Assignee
Centre National de la Recherche Scientifique CNRS
Institut National de la Sante et de la Recherche Medicale INSERM
Sorbonne Universite
Institut du Cerveau et de La Moelle Epiniere ICM
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Publication of CN114555813A publication Critical patent/CN114555813A/zh
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CN202080051431.4A 2019-05-21 2020-05-20 治疗雷特综合征的胆固醇24-水解酶的表达载体 Pending CN114555813A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP19175767.3 2019-05-21
EP19175767 2019-05-21
PCT/EP2020/064092 WO2020234363A2 (en) 2019-05-21 2020-05-20 Expression vector for cholesterol 24-hydrolase in therapy of rett syndrome

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CN114555813A true CN114555813A (zh) 2022-05-27

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US (1) US20220233654A1 (https=)
EP (1) EP3972631B1 (https=)
JP (1) JP7624410B2 (https=)
CN (1) CN114555813A (https=)
WO (1) WO2020234363A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20250262247A1 (en) * 2022-04-20 2025-08-21 The Regents Of The University Of California Microglial cells as therapeutic targets in neurodevelopmental disorders

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US20140179770A1 (en) * 2012-12-12 2014-06-26 Massachusetts Institute Of Technology Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
CN105682688A (zh) * 2013-06-26 2016-06-15 雷特综合征研究信托 Rett综合征及其治疗
US20160175462A1 (en) * 2013-06-17 2016-06-23 The Broad Institute Inc. Delivery, use and therapeutic applications of the crispr-cas systems and compositions for targeting disorders and diseases using viral components
US20180265927A1 (en) * 2004-02-17 2018-09-20 The Hospital For Sick Children Mecp2e1 gene

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WO2020234363A2 (en) 2020-11-26
WO2020234363A8 (en) 2021-10-07
CA3140809A1 (en) 2020-11-26
EP3972631A2 (en) 2022-03-30
US20220233654A1 (en) 2022-07-28
EP3972631B1 (en) 2024-07-10
WO2020234363A3 (en) 2021-11-18
JP2022534202A (ja) 2022-07-28
EP3972631C0 (en) 2024-07-10
JP7624410B2 (ja) 2025-01-30

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