JP2021522836A5 - - Google Patents

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Publication number
JP2021522836A5
JP2021522836A5 JP2020563947A JP2020563947A JP2021522836A5 JP 2021522836 A5 JP2021522836 A5 JP 2021522836A5 JP 2020563947 A JP2020563947 A JP 2020563947A JP 2020563947 A JP2020563947 A JP 2020563947A JP 2021522836 A5 JP2021522836 A5 JP 2021522836A5
Authority
JP
Japan
Prior art keywords
viral vector
vector according
promoter
nucleic acid
rna
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.)
Pending
Application number
JP2020563947A
Other languages
English (en)
Japanese (ja)
Other versions
JP2021522836A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2019/032423 external-priority patent/WO2019222354A1/en
Publication of JP2021522836A publication Critical patent/JP2021522836A/ja
Publication of JP2021522836A5 publication Critical patent/JP2021522836A5/ja
Pending legal-status Critical Current

Links

JP2020563947A 2018-05-15 2019-05-15 スプライスオパシーの軽減、及びｒｎａドミナンス障害の治療のための組成物及び方法 Pending JP2021522836A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862671769P 2018-05-15 2018-05-15
US62/671,769 2018-05-15
PCT/US2019/032423 WO2019222354A1 (en) 2018-05-15 2019-05-15 Compositions and methods for reducing spliceopathy and treating rna dominance disorders

Publications (2)

Publication Number Publication Date
JP2021522836A JP2021522836A (ja) 2021-09-02
JP2021522836A5 true JP2021522836A5 (https=) 2022-05-24

Family

ID=68540673

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2020563947A Pending JP2021522836A (ja) 2018-05-15 2019-05-15 スプライスオパシーの軽減、及びｒｎａドミナンス障害の治療のための組成物及び方法

Country Status (15)

Country Link
US (1) US20210269825A1 (https=)
EP (1) EP3793566A4 (https=)
JP (1) JP2021522836A (https=)
KR (1) KR20210010549A (https=)
CN (1) CN112469421A (https=)
AU (1) AU2019268346A1 (https=)
BR (1) BR112020023298A2 (https=)
CA (1) CA3098249A1 (https=)
CL (1) CL2020002955A1 (https=)
CO (1) CO2020015239A2 (https=)
MA (1) MA51938B1 (https=)
MX (1) MX2020012269A (https=)
PH (1) PH12020551913A1 (https=)
SG (1) SG11202011151VA (https=)
WO (1) WO2019222354A1 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN113166208B (zh) 2018-10-02 2024-11-22 沃雅戈治疗公司 重新定向aav衣壳的嗜性
TWI900553B (zh) 2020-05-13 2025-10-11 美商航海家醫療公司 Aav蛋白殼之趨性重定向
WO2023034870A2 (en) 2021-09-01 2023-03-09 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing dmpk expression
EP4426716A1 (en) 2021-11-02 2024-09-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2023196862A1 (en) * 2022-04-06 2023-10-12 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
US20250352672A1 (en) * 2022-05-13 2025-11-20 University Of Washington Method for treatment of myotonic dystrophy combining protein expression and rna interference vector delivery with tissue detargeting
TW202409289A (zh) * 2022-06-27 2024-03-01 美商安斯泰來基因治療股份有限公司 用於治療肌強直性營養不良之組合物及方法
US20240384271A1 (en) * 2023-04-05 2024-11-21 Genzyme Corporation Targeted gene therapy for dm-1 myotonic dystrophy
WO2024229164A2 (en) * 2023-05-03 2024-11-07 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025038795A1 (en) * 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025122536A1 (en) * 2023-12-05 2025-06-12 Voyager Therapeutics, Inc. Compositions and methods for the treatment of disorders related to dystrophia myotonica protein kinase
WO2025259747A2 (en) * 2024-06-12 2025-12-18 Alnylam Pharmaceuticals, Inc. Dystrophy myotonic protein kinase (dmpk) irna compositions and methods of use thereof

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5977333A (en) * 1992-02-06 1999-11-02 Massachusetts Institute Of Technology DNA sequence encoding the myotonic dystrophy gene and uses thereof
US20050042646A1 (en) * 2002-08-05 2005-02-24 Davidson Beverly L. RNA interference suppresion of neurodegenerative diseases and methods of use thereof
US20120322861A1 (en) * 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
EP2595663A4 (en) * 2010-07-19 2014-03-05 Isis Pharmaceuticals Inc MODULATION OF DYSTROPHIA MYOTONICA PROTEIN KINASE (DMPK) EXPRESSION
HRP20190992T1 (hr) * 2014-12-24 2019-09-20 Uniqure Ip B.V. Suprimiranje gena za huntingtin inducirano s rnai
WO2017079291A1 (en) * 2015-11-02 2017-05-11 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating c90rf72
CN115896106A (zh) * 2016-03-01 2023-04-04 佛罗里达大学研究基金会有限公司 用于治疗显性视网膜色素变性的aav载体
CN109862785B (zh) * 2016-09-30 2022-09-06 瑞泽恩制药公司 C9orf72基因座中具有六核苷酸重复扩增的非人类动物
CN112292895B (zh) * 2018-08-10 2024-06-14 富士通株式会社 一种通信方法、装置和通信系统

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