JP7856581B2 - 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

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Publication number
JP7856581B2
JP7856581B2 JP2022568520A JP2022568520A JP7856581B2 JP 7856581 B2 JP7856581 B2 JP 7856581B2 JP 2022568520 A JP2022568520 A JP 2022568520A JP 2022568520 A JP2022568520 A JP 2022568520A JP 7856581 B2 JP7856581 B2 JP 7856581B2
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sequence
cells
base editing
guide rna
hbg1
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JP2022568520A
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Japanese (ja)
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JP2023526007A (ja
JP2023526007A5 (enExample
Inventor
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 - Hôpitaux de Paris
Fondation Imagine
Université Paris Cité
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Assistance Publique - Hôpitaux de Paris
Fondation Imagine
Université Paris Cité
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Priority to JP2026028532A priority Critical patent/JP2026124805A/ja
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    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
    • C12N15/102—Mutagenizing nucleic acids
    • C—CHEMISTRY; METALLURGY
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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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]
    • C—CHEMISTRY; METALLURGY
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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)
    • C—CHEMISTRY; METALLURGY
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    • C12Y—ENZYMES
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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]
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy
    • C—CHEMISTRY; METALLURGY
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    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/34—Allele or polymorphism specific uses

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Mycology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (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 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)

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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)

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

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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

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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

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

Family Cites Families (8)

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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
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

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，2019年07月22日，Vol. 37, No. 9，p.1070-1079
GabriellaE. MARTYN et al.，Blood，2019年02月21日，Vol. 133, No. 8，p.852-856
LirenWANG et al.，Cell Research，2020年01月07日，Vol. 30, No. 3，p.276-278
Luke WKOBLAN et al.，Nature Biotechnology，2018年10月，Vol. 36, No. 9，p.843-846

Also Published As

Publication number Publication date
JP2023526007A (ja) 2023-06-20
US20230279438A1 (en) 2023-09-07
WO2021228944A1 (en) 2021-11-18
EP4150081A1 (en) 2023-03-22
JP2026124805A (ja) 2026-07-31

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