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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JPWO2022016055A5 (https=
JP2023534293A5 (https=
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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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    • A—HUMAN NECESSITIES
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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)

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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
JPWO2022016055A5 JPWO2022016055A5 (https=) 2024-07-24
JP2023534293A5 JP2023534293A5 (https=) 2024-07-24

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

Families Citing this family (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20250195497A1 (en) * 2022-03-22 2025-06-19 Kyoto University 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
AU2024313758A1 (en) 2023-06-23 2026-01-08 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
US12421524B2 (en) * 2018-09-24 2025-09-23 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: 0005660142 *

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

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