JP2023526007A - 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 - Google Patents

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 Download PDF

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JP2023526007A
JP2023526007A JP2022568520A JP2022568520A JP2023526007A JP 2023526007 A JP2023526007 A JP 2023526007A JP 2022568520 A JP2022568520 A JP 2022568520A JP 2022568520 A JP2022568520 A JP 2022568520A JP 2023526007 A JP2023526007 A JP 2023526007A
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editing
base
cells
promoter
hbg1
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JP2023526007A5 (https=
JPWO2021228944A5 (https=
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ミッチョ，アンナリタ
アントニウ，パナヨティス
カヴァッツァーナ，マリナ
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Assistance Publique Hopitaux de Paris APHP
Fondation Imagine
Universite Paris Cite
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Assistance Publique Hopitaux de Paris APHP
Fondation Imagine
Universite Paris Cite
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Publication of JP2023526007A5 publication Critical patent/JP2023526007A5/ja
Publication of JPWO2021228944A5 publication Critical patent/JPWO2021228944A5/ja
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    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
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    • C12N15/09—Recombinant DNA-technology
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/16—Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22—Ribonucleases [RNase]; Deoxyribonucleases [DNase]
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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    • C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
    • C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
    • C12Y305/04005—Cytidine deaminase (3.5.4.5)
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
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    • C12N2320/00—Applications; Uses
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    • C12N2320/34—Allele or polymorphism specific uses

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  • Crystallography & Structural Chemistry (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
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 Pending JP2023526007A (ja)

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

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JP2023526007A true JP2023526007A (ja) 2023-06-20
JP2023526007A5 JP2023526007A5 (https=) 2024-05-01
JPWO2021228944A5 JPWO2021228944A5 (https=) 2024-05-01

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US (1) US20230279438A1 (https=)
EP (1) EP4150081A1 (https=)
JP (1) JP2023526007A (https=)
WO (1) WO2021228944A1 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN121555430A (zh) 2018-05-11 2026-02-24 比姆医疗股份有限公司 使用可编程碱基编辑器系统取代病原性氨基酸的方法
US20250161492A1 (en) * 2022-01-25 2025-05-22 Institut National de la Santé et de la Recherche Médicale Base editing approaches for the treatment of beta-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
WO2025017033A1 (en) 2023-07-17 2025-01-23 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
WO2025065716A1 (en) * 2023-09-29 2025-04-03 Yoltech Therapeutics Co., Ltd Lipid nanoparticle formulations and methods of use thereof
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

Citations (1)

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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

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WO2008141248A2 (en) 2007-05-10 2008-11-20 Agilent Technologies, Inc. Thiocarbon-protecting groups for rna synthesis
RU2650811C2 (ru) * 2012-02-24 2018-04-17 Фред Хатчинсон Кэнсер Рисерч Сентер Композиции и способы лечения гемоглобинопатии
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
WO2019178426A1 (en) * 2018-03-14 2019-09-19 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
US20220033856A1 (en) * 2018-09-11 2022-02-03 Université de Paris Methods for increasing fetal hemoglobin content in eukaryotic cells and uses thereof for the treatment of hemoglobinopathies

Patent Citations (1)

* 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

Non-Patent Citations (4)

* Cited by examiner, † Cited by third party
Title
B W.THURONYI ET AL., NATURE BIOTECHNOLOGY, vol. 37, no. 9, JPN6025016439, 22 July 2019 (2019-07-22), pages 1070 - 1079, ISSN: 0005717468 *
GABRIELLAE. MARTYN ET AL., BLOOD, vol. 133, no. 8, JPN6025016433, 21 February 2019 (2019-02-21), pages 852 - 856, ISSN: 0005717466 *
LIRENWANG ET AL., CELL RESEARCH, vol. 30, no. 3, JPN6025016432, 7 January 2020 (2020-01-07), pages 276 - 278, ISSN: 0005583747 *
LUKE WKOBLAN ET AL., NATURE BIOTECHNOLOGY, vol. 36, no. 9, JPN6025016435, October 2018 (2018-10-01), pages 843 - 846, ISSN: 0005717467 *

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US20230279438A1 (en) 2023-09-07
EP4150081A1 (en) 2023-03-22
WO2021228944A1 (en) 2021-11-18

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