JPWO2023099591A5 - - Google Patents

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Publication number
JPWO2023099591A5
JPWO2023099591A5 JP2024532454A JP2024532454A JPWO2023099591A5 JP WO2023099591 A5 JPWO2023099591 A5 JP WO2023099591A5 JP 2024532454 A JP2024532454 A JP 2024532454A JP 2024532454 A JP2024532454 A JP 2024532454A JP WO2023099591 A5 JPWO2023099591 A5 JP WO2023099591A5
Authority
JP
Japan
Prior art keywords
seq
guide rna
rna molecule
gene editing
enzyme
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
JP2024532454A
Other languages
English (en)
Japanese (ja)
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JP2024543966A5 (https=
JP2024543966A (ja
Publication date
Application filed filed Critical
Priority claimed from PCT/EP2022/083904 external-priority patent/WO2023099591A1/en
Publication of JP2024543966A publication Critical patent/JP2024543966A/ja
Publication of JPWO2023099591A5 publication Critical patent/JPWO2023099591A5/ja
Publication of JP2024543966A5 publication Critical patent/JP2024543966A5/ja
Pending legal-status Critical Current

Links

JP2024532454A 2021-12-01 2022-11-30 赤血球特異的BCL11Aエンハンサーの+55kb領域を編集することにより胎児ヘモグロビン含量を増加させるための方法。 Pending JP2024543966A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP21306681.4 2021-12-01
EP21306681 2021-12-01
PCT/EP2022/083904 WO2023099591A1 (en) 2021-12-01 2022-11-30 Methods for increasing fetal hemoglobin content by editing the +55-kb region of the erythroid-specific bcl11a enhancer

Publications (3)

Publication Number Publication Date
JP2024543966A JP2024543966A (ja) 2024-11-26
JPWO2023099591A5 true JPWO2023099591A5 (https=) 2025-11-17
JP2024543966A5 JP2024543966A5 (https=) 2025-11-17

Family

ID=79185911

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024532454A Pending JP2024543966A (ja) 2021-12-01 2022-11-30 赤血球特異的BCL11Aエンハンサーの+55kb領域を編集することにより胎児ヘモグロビン含量を増加させるための方法。

Country Status (3)

Country Link
EP (1) EP4441089A1 (https=)
JP (1) JP2024543966A (https=)
WO (1) WO2023099591A1 (https=)

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008141248A2 (en) 2007-05-10 2008-11-20 Agilent Technologies, Inc. Thiocarbon-protecting groups for rna synthesis
EP3241902B1 (en) 2012-05-25 2018-02-28 The Regents of The University of California Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
AU2014227653B2 (en) 2013-03-15 2017-04-20 The General Hospital Corporation Using RNA-guided foki nucleases (RFNs) to increase specificity for RNA-guided genome editing
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
JP7109784B2 (ja) 2015-10-23 2022-08-01 プレジデント アンド フェローズ オブ ハーバード カレッジ 遺伝子編集のための進化したCas9蛋白質
KR102547316B1 (ko) 2016-08-03 2023-06-23 프레지던트 앤드 펠로우즈 오브 하바드 칼리지 아데노신 핵염기 편집제 및 그의 용도
KR20210139265A (ko) 2019-02-13 2021-11-22 빔 테라퓨틱스, 인크. 표적 서열에서 핵염기를 변형하기 위한 아데노신 데아미나제 염기 편집기 및 이의 사용 방법
CN114667149A (zh) 2019-09-09 2022-06-24 比姆医疗股份有限公司 新型核碱基编辑器及其使用方法
WO2021067233A1 (en) * 2019-09-30 2021-04-08 The Children's Medical Center Corporation Bcl11a guide and base editor delivery

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