JP2022534202A5 - - Google Patents

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Publication number
JP2022534202A5
JP2022534202A5 JP2021568858A JP2021568858A JP2022534202A5 JP 2022534202 A5 JP2022534202 A5 JP 2022534202A5 JP 2021568858 A JP2021568858 A JP 2021568858A JP 2021568858 A JP2021568858 A JP 2021568858A JP 2022534202 A5 JP2022534202 A5 JP 2022534202A5
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JP
Japan
Prior art keywords
vector
treatment
rtt
neurons
rtt according
Prior art date
Legal status (The legal status 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 status listed.)
Granted
Application number
JP2021568858A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022534202A (ja
JP7624410B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/EP2020/064092 external-priority patent/WO2020234363A2/en
Publication of JP2022534202A publication Critical patent/JP2022534202A/ja
Publication of JP2022534202A5 publication Critical patent/JP2022534202A5/ja
Application granted granted Critical
Publication of JP7624410B2 publication Critical patent/JP7624410B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2021568858A 2019-05-21 2020-05-20 レット症候群の治療におけるコレステロール 24-ヒドロラーゼのための発現ベクター Active JP7624410B2 (ja)

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

Publications (3)

Publication Number Publication Date
JP2022534202A JP2022534202A (ja) 2022-07-28
JP2022534202A5 true JP2022534202A5 (https=) 2023-05-31
JP7624410B2 JP7624410B2 (ja) 2025-01-30

Family

ID=66751865

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2021568858A Active JP7624410B2 (ja) 2019-05-21 2020-05-20 レット症候群の治療におけるコレステロール 24-ヒドロラーゼのための発現ベクター

Country Status (5)

Country Link
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

Family Cites Families (24)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US4861719A (en) 1986-04-25 1989-08-29 Fred Hutchinson Cancer Research Center DNA constructs for retrovirus packaging cell lines
US5278056A (en) 1988-02-05 1994-01-11 The Trustees Of Columbia University In The City Of New York Retroviral packaging cell lines and process of using same
US5670488A (en) 1992-12-03 1997-09-23 Genzyme Corporation Adenovirus vector for gene therapy
AU8200191A (en) 1990-07-09 1992-02-04 United States of America, as represented by the Secretary, U.S. Department of Commerce, The High efficiency packaging of mutant adeno-associated virus using amber suppressions
US5173414A (en) 1990-10-30 1992-12-22 Applied Immune Sciences, Inc. Production of recombinant adeno-associated virus vectors
DE69233013T2 (de) 1991-08-20 2004-03-04 The Government Of The United States Of America As Represented By The Secretary Of National Institute Of Health, Office Of Technology Transfer Adenovirus vermittelter gentransfer in den gastrointestinaltrakt
DE69434860T2 (de) 1993-02-22 2007-03-15 The Rockefeller University Herstellung von helfer-freien retroviren mit hohem titer mittels transienter transfektion
FR2712812B1 (fr) 1993-11-23 1996-02-09 Centre Nat Rech Scient Composition pour la production de produits thérapeutiques in vivo.
IL116816A (en) 1995-01-20 2003-05-29 Rhone Poulenc Rorer Sa Cell for the production of a defective recombinant adenovirus or an adeno-associated virus and the various uses thereof
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
CA2554380C (en) * 2004-02-17 2015-05-19 The Hospital For Sick Children Mecp2e1 gene
EP2187898B8 (en) * 2007-09-12 2016-09-14 INSERM (Institut National de la Santé et de la Recherche Médicale) Use of viral vectors carrying the cyp46a1 gene for the treatment of alzheimer's disease
US9132173B2 (en) 2010-10-15 2015-09-15 Institut National De La Sante Et De La Recherche Medicale (Inserm) Expression vector for cholesterol 24-hydrolase in therapy of Huntington's disease
EP3031921B1 (en) * 2012-12-12 2025-03-12 The Broad Institute, Inc. Delivery, engineering and optimization of systems, methods and compositions for sequence manipulation and therapeutic applications
MX374532B (es) * 2013-06-17 2025-03-06 Broad Inst Inc Suministro, uso y aplicaciones terapéuticas de los sistemas y composiciones crispr-cas, para actuar sobre trastornos y enfermedades utilizando componentes víricos.
WO2014210389A1 (en) * 2013-06-26 2014-12-31 Rett Syndrome Research Trust Rett syndrome and treatments therefore
ES2739288T3 (es) 2013-09-13 2020-01-30 California Inst Of Techn Recuperación selectiva
EP3151866B1 (en) 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
CA2962406A1 (en) * 2014-09-25 2016-03-31 Cold Spring Harbor Laboratory Treatment of rett syndrome
KR102423442B1 (ko) 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
JP7618386B2 (ja) * 2017-01-30 2025-01-21 ブレインヴェクティス ポリグルタミン反復脊髄小脳失調症の処置におけるコレステロール24-ヒドロキシラーゼ(hydrolase)のための発現ベクター
EP3388520A1 (en) * 2017-04-11 2018-10-17 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for reducing the expression of nkcc1 in a subject in need thereof
US11680275B2 (en) * 2017-06-06 2023-06-20 University Of Massachusetts Self-regulating AAV vectors for safe expression of MeCP2 in rett syndrome

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