JP2024533312A5 - - Google Patents

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Publication number
JP2024533312A5
JP2024533312A5 JP2024515068A JP2024515068A JP2024533312A5 JP 2024533312 A5 JP2024533312 A5 JP 2024533312A5 JP 2024515068 A JP2024515068 A JP 2024515068A JP 2024515068 A JP2024515068 A JP 2024515068A JP 2024533312 A5 JP2024533312 A5 JP 2024533312A5
Authority
JP
Japan
Prior art keywords
sequence
grna
template rna
mutation
gene
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
JP2024515068A
Other languages
English (en)
Japanese (ja)
Other versions
JP2024533312A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2022/076073 external-priority patent/WO2023039447A2/en
Publication of JP2024533312A publication Critical patent/JP2024533312A/ja
Publication of JP2024533312A5 publication Critical patent/JP2024533312A5/ja
Pending legal-status Critical Current

Links

JP2024515068A 2021-09-08 2022-09-07 Serpina調節組成物及び方法 Pending JP2024533312A (ja)

Applications Claiming Priority (7)

Application Number Priority Date Filing Date Title
US202163241970P 2021-09-08 2021-09-08
US63/241,970 2021-09-08
US202163253087P 2021-10-06 2021-10-06
US63/253,087 2021-10-06
US202263303905P 2022-01-27 2022-01-27
US63/303,905 2022-01-27
PCT/US2022/076073 WO2023039447A2 (en) 2021-09-08 2022-09-07 Serpina-modulating compositions and methods

Publications (2)

Publication Number Publication Date
JP2024533312A JP2024533312A (ja) 2024-09-12
JP2024533312A5 true JP2024533312A5 (https=) 2025-09-17

Family

ID=85506923

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024515068A Pending JP2024533312A (ja) 2021-09-08 2022-09-07 Serpina調節組成物及び方法

Country Status (7)

Country Link
US (1) US20240084334A1 (https=)
EP (1) EP4399309A4 (https=)
JP (1) JP2024533312A (https=)
AU (1) AU2022344251A1 (https=)
CA (1) CA3231594A1 (https=)
MX (1) MX2024002988A (https=)
WO (1) WO2023039447A2 (https=)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20150165054A1 (en) 2013-12-12 2015-06-18 President And Fellows Of Harvard College Methods for correcting caspase-9 point mutations
EP3844272A1 (en) 2018-08-28 2021-07-07 Flagship Pioneering Innovations VI, LLC Methods and compositions for modulating a genome
CN116209756A (zh) 2020-03-04 2023-06-02 旗舰先锋创新Vi有限责任公司 调控基因组的方法和组合物
KR20230053735A (ko) 2020-03-04 2023-04-21 플래그쉽 파이어니어링 이노베이션스 브이아이, 엘엘씨 게놈의 조정을 위한 개선된 방법 및 조성물
IL297761A (en) 2020-05-08 2022-12-01 Broad Inst Inc Methods and compositions for simultaneously editing two helices of a designated double-helix nucleotide sequence
CA3231712A1 (en) 2021-09-08 2023-03-16 Flagship Pioneering Innovations Vi, Llc Pah-modulating compositions and methods
AU2022343268A1 (en) 2021-09-08 2024-03-28 Flagship Pioneering Innovations Vi, Llc Methods and compositions for modulating a genome
WO2023108153A2 (en) 2021-12-10 2023-06-15 Flagship Pioneering Innovations Vi, Llc Cftr-modulating compositions and methods
WO2025155772A1 (en) * 2024-01-19 2025-07-24 Gene Company (Pty)Ltd. Alpha-1 anti-trypsin upregulating polynucleotides and method of use and treatment thereof
US20250269059A1 (en) * 2024-02-27 2025-08-28 Crispr Therapeutics Ag Rt editing compositions and methods
WO2025194124A1 (en) * 2024-03-14 2025-09-18 Tessera Therapeutics, Inc. Modified st1cas9 guide nucleic acids
US20250354138A1 (en) * 2024-03-15 2025-11-20 Beam Therapeutics Inc. Prime editing of single base mutations in alpha-1 antitrypsin deficiency

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9458457B2 (en) * 2013-07-03 2016-10-04 Dicerna Pharmaceuticals, Inc. Methods and compositions for the specific inhibition of alpha-1 antitrypsin by double-stranded RNA
CN109715801B (zh) * 2015-12-01 2022-11-01 克里斯普治疗股份公司 用于治疗α1抗胰蛋白酶缺乏的材料和方法
IL308824A (en) * 2016-08-17 2024-01-01 Factor Bioscience Inc Nucleic acid products and methods of administration thereof
CA3035534A1 (en) * 2016-09-07 2018-03-15 Sangamo Therapeutics, Inc. Modulation of liver genes
WO2020191233A1 (en) * 2019-03-19 2020-09-24 The Broad Institute, Inc. Methods and compositions for editing nucleotide sequences
WO2020214609A1 (en) * 2019-04-14 2020-10-22 Duke University Aav vector-mediated deletion of large mutational hotspot for treatment of duchenne muscular dystrophy
US12435330B2 (en) * 2019-10-10 2025-10-07 The Broad Institute, Inc. Methods and compositions for prime editing RNA

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