JP2023526007A5 - - Google Patents

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Publication number
JP2023526007A5
JP2023526007A5 JP2022568520A JP2022568520A JP2023526007A5 JP 2023526007 A5 JP2023526007 A5 JP 2023526007A5 JP 2022568520 A JP2022568520 A JP 2022568520A JP 2022568520 A JP2022568520 A JP 2022568520A JP 2023526007 A5 JP2023526007 A5 JP 2023526007A5
Authority
JP
Japan
Prior art keywords
hbg1
promoter
gene editing
hbg2
eukaryotic cells
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.)
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Application number
JP2022568520A
Other languages
English (en)
Japanese (ja)
Other versions
JP2023526007A (ja
JP7856581B2 (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/EP2021/062633 external-priority patent/WO2021228944A1/en
Publication of JP2023526007A publication Critical patent/JP2023526007A/ja
Publication of JP2023526007A5 publication Critical patent/JP2023526007A5/ja
Priority to JP2026028532A priority Critical patent/JP2026124805A/ja
Application granted granted Critical
Publication of JP7856581B2 publication Critical patent/JP7856581B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2022568520A 2020-05-13 2021-05-12 β-ヘモグロビン異常症の処置のための塩基編集アプローチ Active JP7856581B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2026028532A JP2026124805A (ja) 2020-05-13 2026-02-25 β-ヘモグロビン異常症の処置のための塩基編集アプローチ

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP20305488.7 2020-05-13
EP20305488 2020-05-13
PCT/EP2021/062633 WO2021228944A1 (en) 2020-05-13 2021-05-12 Base editing approaches for the treatment of betahemoglobinopathies

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2026028532A Division JP2026124805A (ja) 2020-05-13 2026-02-25 β-ヘモグロビン異常症の処置のための塩基編集アプローチ

Publications (3)

Publication Number Publication Date
JP2023526007A JP2023526007A (ja) 2023-06-20
JP2023526007A5 true JP2023526007A5 (enExample) 2024-05-01
JP7856581B2 JP7856581B2 (ja) 2026-05-11

Family

ID=71575241

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022568520A Active JP7856581B2 (ja) 2020-05-13 2021-05-12 β-ヘモグロビン異常症の処置のための塩基編集アプローチ
JP2026028532A Pending JP2026124805A (ja) 2020-05-13 2026-02-25 β-ヘモグロビン異常症の処置のための塩基編集アプローチ

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2026028532A Pending JP2026124805A (ja) 2020-05-13 2026-02-25 β-ヘモグロビン異常症の処置のための塩基編集アプローチ

Country Status (4)

Country Link
US (1) US20230279438A1 (enExample)
EP (1) EP4150081A1 (enExample)
JP (2) JP7856581B2 (enExample)
WO (1) WO2021228944A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019217942A1 (en) 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
WO2023144104A1 (en) * 2022-01-25 2023-08-03 INSERM (Institut National de la Santé et de la Recherche Médicale) Base editing approaches for the treatment of βeta-thalassemia
WO2025017030A1 (en) 2023-07-17 2025-01-23 Institut National de la Santé et de la Recherche Médicale Prime editing of the -200 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell
EP4747377A1 (en) 2023-07-17 2026-05-27 Institut National de la Santé et de la Recherche Médicale Prime editing of the -115 region in the hbg1 and/or hbg2 promoter for increasing fetal hemoglobin content in a eukaryotic cell
CN122341362A (zh) * 2023-09-29 2026-07-03 上海尧唐生物科技股份有限公司 脂质纳米颗粒制剂及其使用方法
WO2025120664A1 (en) * 2023-12-04 2025-06-12 Christian Medical College Strategies for precision editing of homologous regions
WO2025147212A1 (en) * 2024-01-05 2025-07-10 Singapore Health Services Pte Ltd Induction of fetal haemoglobin using paired cas9 nickases
WO2026069358A1 (en) * 2024-09-24 2026-04-02 Christian Medical College Base editing methods and compositions

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2476689T3 (pl) 2007-05-10 2016-04-29 Agilent Technologies Inc Tiowęglowe grupy zabezpieczające do syntezy RNA
EP2836226B1 (en) * 2012-02-24 2017-06-28 Fred Hutchinson Cancer Research Center Compositions and methods for the treatment of hemoglobinopathies
ES2728782T3 (es) 2012-05-25 2019-10-28 Univ California Métodos y composiciones para la modificación de ADN objetivo dirigida por ARN y para la modulación de la transcripción dirigida por ARN
US9234213B2 (en) 2013-03-15 2016-01-12 System Biosciences, Llc Compositions and methods directed to CRISPR/Cas genomic engineering systems
CN105408483A (zh) 2013-03-15 2016-03-16 通用医疗公司 遗传和表观遗传调节蛋白至特定基因组基因座的rna引导的靶向
US20140273230A1 (en) 2013-03-15 2014-09-18 Sigma-Aldrich Co., Llc Crispr-based genome modification and regulation
IL277234B2 (en) * 2018-03-14 2026-03-01 Editas Medicine Inc Systems and methods for treating hemoglobin diseases
WO2019217942A1 (en) * 2018-05-11 2019-11-14 Beam Therapeutics Inc. Methods of substituting pathogenic amino acids using programmable base editor systems
EP3850094A1 (en) * 2018-09-11 2021-07-21 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods for increasing fetal hemoglobin content in eukaryotic cells and uses thereof for the treatment of hemoglobinopathies

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