JP2023534293A - 脆弱ｘ症候群の治療のための方法及び組成物 - Google Patents

脆弱ｘ症候群の治療のための方法及び組成物 Download PDF

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JP2023534293A
JP2023534293A JP2023503074A JP2023503074A JP2023534293A JP 2023534293 A JP2023534293 A JP 2023534293A JP 2023503074 A JP2023503074 A JP 2023503074A JP 2023503074 A JP2023503074 A JP 2023503074A JP 2023534293 A JP2023534293 A JP 2023534293A
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JP2023534293A5 (OSRAM
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ペダパティ，アーネスト
グロス，クリスティーナ
エリクソン，クレイグ
ディスミューク，デイビッド
シルバ，エランディ カンチャナ デ
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Cincinnati Childrens Hospital Medical Center
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    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • C12N2830/38—Vector systems having a special element relevant for transcription being a stuffer
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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JP2023503074A 2020-07-17 2021-07-16 脆弱ｘ症候群の治療のための方法及び組成物 Pending JP2023534293A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063053461P 2020-07-17 2020-07-17
US63/053,461 2020-07-17
PCT/US2021/041975 WO2022016055A1 (en) 2020-07-17 2021-07-16 Methods and compositions for treatment of fragile x syndrome

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JP2023534293A true JP2023534293A (ja) 2023-08-08
JP2023534293A5 JP2023534293A5 (OSRAM) 2024-07-24

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US (1) US20230295654A1 (OSRAM)
EP (1) EP4181877A4 (OSRAM)
JP (1) JP2023534293A (OSRAM)
KR (1) KR20230084465A (OSRAM)
CN (1) CN117136077A (OSRAM)
AU (1) AU2021308666A1 (OSRAM)
CA (1) CA3189657A1 (OSRAM)
IL (1) IL299889A (OSRAM)
WO (1) WO2022016055A1 (OSRAM)

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4497436A4 (en) * 2022-03-22 2025-08-06 Univ Kyoto THERAPEUTIC OR PROPHYLACTIC MEDICINE FOR FRAGILE X SYNDROME
GB202206336D0 (en) * 2022-04-29 2022-06-15 Univ Edinburgh Recombinant therapeutic FMR1 constructs and methods of treating fragile X syndrome and related disorders
EP4731267A1 (en) 2023-06-23 2026-04-29 uniQure biopharma B.V. Novel fragile x constructs

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013544511A (ja) * 2010-10-28 2013-12-19 ナノドク リミテッド 特異的内在性ｍｉＲＮＡにより発現を活性化する組成物および方法
US20180311290A1 (en) * 2015-04-23 2018-11-01 University Of Massachusetts Modulation of aav vector transgene expression
WO2019227219A1 (en) * 2018-05-31 2019-12-05 The Governing Council Of The University Of Toronto Adeno-associated viral vector-mediated gene therapy for treating fragile x-associated disorders

Family Cites Families (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3856910B1 (en) * 2018-09-24 2024-11-20 Merck Sharp & Dohme LLC Expression vectors for eukaryotic expression systems

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2013544511A (ja) * 2010-10-28 2013-12-19 ナノドク リミテッド 特異的内在性ｍｉＲＮＡにより発現を活性化する組成物および方法
US20180311290A1 (en) * 2015-04-23 2018-11-01 University Of Massachusetts Modulation of aav vector transgene expression
WO2019227219A1 (en) * 2018-05-31 2019-12-05 The Governing Council Of The University Of Toronto Adeno-associated viral vector-mediated gene therapy for treating fragile x-associated disorders

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
BRAIN SCI, vol. vol. 9(2), JPN6025032681, 2 February 2019 (2019-02-02), pages 32 - 1, ISSN: 0005660143 *
GENE THER, vol. vol. 27(6), JPN6025032682, 12 March 2020 (2020-03-12), pages 247 - 253, ISSN: 0005660144 *
NUCLEIC ACIDS RES, vol. vol. 44(8), JPN6025032680, 25 February 2016 (2016-02-25), pages 3865 - 3877, ISSN: 0005846200 *

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EP4181877A4 (en) 2024-10-23
IL299889A (en) 2023-03-01
CN117136077A (zh) 2023-11-28
WO2022016055A1 (en) 2022-01-20
AU2021308666A1 (en) 2023-02-16
KR20230084465A (ko) 2023-06-13
CA3189657A1 (en) 2022-01-20
EP4181877A1 (en) 2023-05-24
US20230295654A1 (en) 2023-09-21

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