JP2024519922A5 - - Google Patents

Info

Publication number
JP2024519922A5
JP2024519922A5 JP2023571978A JP2023571978A JP2024519922A5 JP 2024519922 A5 JP2024519922 A5 JP 2024519922A5 JP 2023571978 A JP2023571978 A JP 2023571978A JP 2023571978 A JP2023571978 A JP 2023571978A JP 2024519922 A5 JP2024519922 A5 JP 2024519922A5
Authority
JP
Japan
Prior art keywords
sequence
nucleic acid
acid encoding
isolated polynucleotide
polynucleotide
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
JP2023571978A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024519922A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/030359 external-priority patent/WO2022246266A1/en
Publication of JP2024519922A publication Critical patent/JP2024519922A/ja
Publication of JP2024519922A5 publication Critical patent/JP2024519922A5/ja
Pending legal-status Critical Current

Links

JP2023571978A 2021-05-21 2022-05-20 トランスサイレチン遺伝子の塩基編集 Pending JP2024519922A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US202163191458P 2021-05-21 2021-05-21
US63/191,458 2021-05-21
US202263322182P 2022-03-21 2022-03-21
US63/322,182 2022-03-21
PCT/US2022/030359 WO2022246266A1 (en) 2021-05-21 2022-05-20 Base editing of transthyretin gene

Publications (2)

Publication Number Publication Date
JP2024519922A JP2024519922A (ja) 2024-05-21
JP2024519922A5 true JP2024519922A5 (enExample) 2025-05-27

Family

ID=84141870

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2023571978A Pending JP2024519922A (ja) 2021-05-21 2022-05-20 トランスサイレチン遺伝子の塩基編集

Country Status (7)

Country Link
US (1) US20240124536A1 (enExample)
EP (1) EP4352216A4 (enExample)
JP (1) JP2024519922A (enExample)
KR (1) KR20240010720A (enExample)
AU (1) AU2022275546A1 (enExample)
CA (1) CA3220738A1 (enExample)
WO (1) WO2022246266A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20260094824A (ko) 2023-10-25 2026-06-23 씨에스피씨 종콰이 팔마씨우티컬 테크놀로지 (스자좡) 컴퍼니 리미티드 고효율, 저오프타겟 유전자 편집 도구

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11866727B2 (en) * 2015-11-06 2024-01-09 Crispr Therapeutics Ag Materials and methods for treatment of glycogen storage disease type 1A
ES3030658T3 (en) * 2016-03-30 2025-07-01 Intellia Therapeutics Inc Lipid nanoparticle formulations for crispr/cas components
WO2018007871A1 (en) * 2016-07-08 2018-01-11 Crispr Therapeutics Ag Materials and methods for treatment of transthyretin amyloidosis
TWI904069B (zh) * 2017-09-29 2025-11-11 美商英特利亞醫療公司 用於ttr基因編輯及治療attr澱粉樣變性之組合物及方法
KR102959497B1 (ko) * 2018-05-11 2026-05-04 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 병원성 돌연변이를 억제하는 방법
KR102853088B1 (ko) * 2018-05-11 2025-09-02 빔 테라퓨틱스, 인크. 프로그래밍가능한 염기 편집기 시스템을 이용하여 단일염기다형성을 편집하는 방법
WO2019226953A1 (en) * 2018-05-23 2019-11-28 The Broad Institute, Inc. Base editors and uses thereof
CN114040970B (zh) * 2019-02-13 2024-09-27 比姆医疗股份有限公司 使用腺苷脱氨酶碱基编辑器编辑疾病相关基因的方法,包括遗传性疾病的治疗
US20220098593A1 (en) * 2019-02-13 2022-03-31 Beam Therapeutics Inc. Splice acceptor site disruption of a disease-associated gene using adenosine deaminase base editors, including for the treatment of genetic disease
KR20220004648A (ko) * 2019-03-28 2022-01-11 인텔리아 테라퓨틱스, 인크. Ttr 가이드 rna, 및 rna-가이드 dna 결합제를 암호화하는 폴리뉴클레오티드를 포함하는 조성물 및 방법
MX2021011690A (es) * 2019-03-28 2022-01-06 Intellia Therapeutics Inc Composiciones y métodos para la edición del gen ttr y el tratamiento de la amiloidosis attr que comprende un corticosteroide o uso de este.
EP4259792A1 (en) * 2020-12-11 2023-10-18 Intellia Therapeutics, Inc. Polynucleotides, compositions, and methods for genome editing involving deamination
CN117729931A (zh) * 2021-05-14 2024-03-19 比姆医疗股份有限公司 用于治疗转甲状腺素蛋白淀粉样变性的组合物和方法

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