JP2022553406A5 - - Google Patents

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Publication number
JP2022553406A5
JP2022553406A5 JP2022524118A JP2022524118A JP2022553406A5 JP 2022553406 A5 JP2022553406 A5 JP 2022553406A5 JP 2022524118 A JP2022524118 A JP 2022524118A JP 2022524118 A JP2022524118 A JP 2022524118A JP 2022553406 A5 JP2022553406 A5 JP 2022553406A5
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JP
Japan
Prior art keywords
sequence
expression cassette
nucleotides
seq
raav
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
JP2022524118A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022553406A (ja
JP7697943B2 (ja
Filing date
Publication date
Priority claimed from PCT/US2019/067872 external-priority patent/WO2020132455A1/en
Application filed filed Critical
Priority claimed from PCT/US2020/056881 external-priority patent/WO2021081217A1/en
Publication of JP2022553406A publication Critical patent/JP2022553406A/ja
Publication of JP2022553406A5 publication Critical patent/JP2022553406A5/ja
Application granted granted Critical
Publication of JP7697943B2 publication Critical patent/JP7697943B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2022524118A 2019-10-23 2020-10-22 導入遺伝子発現のｄｒｇ特異的低減のための組成物 Active JP7697943B2 (ja)

Applications Claiming Priority (17)

Application Number Priority Date Filing Date Title
US201962924970P 2019-10-23 2019-10-23
US62/924,970 2019-10-23
US201962934915P 2019-11-13 2019-11-13
US62/934,915 2019-11-13
USPCT/US2019/067872 2019-12-20
PCT/US2019/067872 WO2020132455A1 (en) 2018-12-21 2019-12-20 Compositions for drg-specific reduction of transgene expression
US202062972404P 2020-02-10 2020-02-10
US62/972,404 2020-02-10
US202063005894P 2020-04-06 2020-04-06
US63/005,894 2020-04-06
US202063023602P 2020-05-12 2020-05-12
US63/023,602 2020-05-12
US202063038514P 2020-06-12 2020-06-12
US63/038,514 2020-06-12
US202063043600P 2020-06-24 2020-06-24
US63/043,600 2020-06-24
PCT/US2020/056881 WO2021081217A1 (en) 2019-10-23 2020-10-22 Compositions for drg-specific reduction of transgene expression

Publications (3)

Publication Number Publication Date
JP2022553406A JP2022553406A (ja) 2022-12-22
JP2022553406A5 true JP2022553406A5 (https=) 2023-10-18
JP7697943B2 JP7697943B2 (ja) 2025-06-24

Family

ID=75620360

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022524118A Active JP7697943B2 (ja) 2019-10-23 2020-10-22 導入遺伝子発現のｄｒｇ特異的低減のための組成物

Country Status (8)

Country Link
US (1) US20220389457A1 (https=)
EP (1) EP4048785A4 (https=)
JP (1) JP7697943B2 (https=)
KR (1) KR20220105158A (https=)
AU (1) AU2020369570A1 (https=)
CA (1) CA3155154A1 (https=)
IL (1) IL292372A (https=)
WO (1) WO2021081217A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2022014258A (es) * 2020-05-12 2023-02-22 Univ Pennsylvania Composiciones para la reducción específica de un fármaco de la expresión de transgén.
CA3190399A1 (en) * 2020-08-24 2022-03-03 James M. Wilson Viral vectors encoding glp-1 receptor agonist fusions and uses thereof in treating metabolic diseases
MX2024004723A (es) * 2021-10-18 2024-07-19 Univ Pennsylvania Composiciones útiles en el tratamiento del trastorno por deficiencia de cdkl5 (cdd).
EP4423264A4 (en) * 2021-10-27 2025-12-17 Univ North Carolina Chapel Hill AAV-IDUA VECTOR FOR TREATING MPS I
EP4551253A1 (en) * 2022-07-08 2025-05-14 Ospedale San Raffaele S.r.l. Transgene cassettes
CN116064593B (zh) * 2023-02-09 2024-05-14 四川大学 一种毛白杨pgag基因及其应用
US20250276095A1 (en) * 2024-03-04 2025-09-04 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013151665A2 (en) * 2012-04-02 2013-10-10 modeRNA Therapeutics Modified polynucleotides for the production of proteins associated with human disease
FR3004463A1 (fr) 2013-04-11 2014-10-17 Genethon Systeme d'expression pour une therapie genique selective
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
HK1256341A1 (zh) 2015-05-15 2019-09-20 明尼苏达大学董事会 用於治疗性递送到中枢神经系统的腺相关物
CA3012195A1 (en) * 2016-02-03 2017-08-10 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type i
CA3011943A1 (en) * 2016-02-22 2017-08-31 The University Of North Carolina At Chapel Hill Aav-idua vector for treatment of mps i-associated blindness
CA3068328A1 (en) 2017-07-06 2019-01-10 The Trustees Of The University Of Pennsylvania Aav9-mediated gene therapy for treating mucopolysaccharidosis type i
RU2770922C2 (ru) * 2017-09-20 2022-04-25 4Д Молекьюлар Терапьютикс Инк. Капсиды вариантов аденоассоциированных вирусов и методы их применения

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