JP2022544004A5 - - Google Patents

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Publication number
JP2022544004A5
JP2022544004A5 JP2022503959A JP2022503959A JP2022544004A5 JP 2022544004 A5 JP2022544004 A5 JP 2022544004A5 JP 2022503959 A JP2022503959 A JP 2022503959A JP 2022503959 A JP2022503959 A JP 2022503959A JP 2022544004 A5 JP2022544004 A5 JP 2022544004A5
Authority
JP
Japan
Prior art keywords
seq
aav
expression cassette
promoter
nucleic acid
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
JP2022503959A
Other languages
English (en)
Japanese (ja)
Other versions
JP2022544004A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/043578 external-priority patent/WO2021021661A1/en
Publication of JP2022544004A publication Critical patent/JP2022544004A/ja
Publication of JP2022544004A5 publication Critical patent/JP2022544004A5/ja
Pending legal-status Critical Current

Links

JP2022503959A 2019-07-26 2020-07-24 操作された核酸調節エレメントならびにその使用方法 Pending JP2022544004A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US201962879358P 2019-07-26 2019-07-26
US62/879,358 2019-07-26
US202062967480P 2020-01-29 2020-01-29
US62/967,480 2020-01-29
US202063023152P 2020-05-11 2020-05-11
US63/023,152 2020-05-11
PCT/US2020/043578 WO2021021661A1 (en) 2019-07-26 2020-07-24 Engineered nucleic acid regulatory element and methods of uses thereof

Publications (2)

Publication Number Publication Date
JP2022544004A JP2022544004A (ja) 2022-10-17
JP2022544004A5 true JP2022544004A5 (https=) 2023-08-02

Family

ID=72047119

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022503959A Pending JP2022544004A (ja) 2019-07-26 2020-07-24 操作された核酸調節エレメントならびにその使用方法

Country Status (6)

Country Link
US (1) US20230042103A1 (https=)
EP (1) EP4004214A1 (https=)
JP (1) JP2022544004A (https=)
CA (1) CA3145112A1 (https=)
IL (1) IL289922A (https=)
WO (1) WO2021021661A1 (https=)

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EP4707401A2 (en) * 2021-04-09 2026-03-11 University of Washington Artificial regulatory cassettes for muscle-specific gene expression
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US20250025574A1 (en) * 2021-10-28 2025-01-23 Regenxbio Inc. Engineered nucleic acid regulatory elements and methods and uses thereof
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CN119213133B (zh) 2022-05-16 2026-01-06 上海天泽云泰生物医药有限公司 用于治疗i型戊二酸尿症的重组aav载体
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KR20260005328A (ko) * 2023-04-28 2026-01-09 청두 오리젠 바이오테크놀로지 씨오., 엘티디. 항vegf 단백질을 코딩하는 핵산, 폴리뉴클레오타이드 발현 카세트 및 재조합 아데노 관련 바이러스
WO2026028089A1 (en) * 2024-07-29 2026-02-05 Novartis Ag Compositions and methods for the treatment of lysosomal acid lipase deficiency (lal-d)

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