JP2024543966A5 - - Google Patents

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Publication number
JP2024543966A5
JP2024543966A5 JP2024532454A JP2024532454A JP2024543966A5 JP 2024543966 A5 JP2024543966 A5 JP 2024543966A5 JP 2024532454 A JP2024532454 A JP 2024532454A JP 2024532454 A JP2024532454 A JP 2024532454A JP 2024543966 A5 JP2024543966 A5 JP 2024543966A5
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JP
Japan
Prior art keywords
seq
guide rna
rna molecule
base 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
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English (en)
Japanese (ja)
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JP2024543966A (ja
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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 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 2021-12-01
EP21306681.4 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 (2)

Publication Number Publication Date
JP2024543966A JP2024543966A (ja) 2024-11-26
JP2024543966A5 true 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
DK2476689T3 (en) 2007-05-10 2016-01-18 Agilent Technologies Inc Thiocarbonbeskyttende groups for RNA synthesis
AU2013266968B2 (en) 2012-05-25 2017-06-29 Emmanuelle CHARPENTIER Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
CN112301024A (zh) 2013-03-15 2021-02-02 通用医疗公司 使用RNA引导的FokI核酸酶(RFN)提高RNA引导的基因组编辑的特异性
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
IL310721B2 (en) 2015-10-23 2025-11-01 Harvard College Nucleobase editors and uses thereof
CN110214183A (zh) 2016-08-03 2019-09-06 哈佛大学的校长及成员们 腺苷核碱基编辑器及其用途
JP7693552B2 (ja) 2019-02-13 2025-06-17 ビーム セラピューティクス インク. アデノシンデアミナーゼ塩基エディターおよびそれを用いて標的配列中の核酸塩基を改変する方法
AU2020344547A1 (en) 2019-09-09 2022-03-24 Beam Therapeutics Inc. Novel nucleobase editors and methods of using same
EP4017980A4 (en) * 2019-09-30 2024-08-07 The Children's Medical Center Corporation BCL11A GUIDE AND BASIC EDITOR DISTRIBUTION

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