CN121844044A - Bcl11a基因的增强子的一部分或全部被破坏了的造血干细胞的制造方法、和用于该方法的试剂盒 - Google Patents

Bcl11a基因的增强子的一部分或全部被破坏了的造血干细胞的制造方法、和用于该方法的试剂盒

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Publication number
CN121844044A
CN121844044A CN202480058255.5A CN202480058255A CN121844044A CN 121844044 A CN121844044 A CN 121844044A CN 202480058255 A CN202480058255 A CN 202480058255A CN 121844044 A CN121844044 A CN 121844044A
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CN
China
Prior art keywords
protein
enhancer
crispr
cas3
hematopoietic stem
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
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CN202480058255.5A
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English (en)
Chinese (zh)
Inventor
小堀峻吾
佐伯凉太
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C4u Corp
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C4u Corp
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Application filed by C4u Corp filed Critical C4u Corp
Publication of CN121844044A publication Critical patent/CN121844044A/zh
Pending legal-status Critical Current

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10Cells modified by introduction of foreign genetic material
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CN202480058255.5A 2023-10-30 2024-10-29 Bcl11a基因的增强子的一部分或全部被破坏了的造血干细胞的制造方法、和用于该方法的试剂盒 Pending CN121844044A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
JP2023185785 2023-10-30
JP2023-185785 2023-10-30
PCT/JP2024/038444 WO2025094917A1 (ja) 2023-10-30 2024-10-29 Bcl11a遺伝子のエンハンサーの一部又は全部が破壊された造血幹細胞の製造方法、及びその方法に用いるためのキット

Publications (1)

Publication Number Publication Date
CN121844044A true CN121844044A (zh) 2026-04-10

Family

ID=95582304

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202480058255.5A Pending CN121844044A (zh) 2023-10-30 2024-10-29 Bcl11a基因的增强子的一部分或全部被破坏了的造血干细胞的制造方法、和用于该方法的试剂盒

Country Status (3)

Country Link
JP (1) JPWO2025094917A1 (https=)
CN (1) CN121844044A (https=)
WO (1) WO2025094917A1 (https=)

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN109554350B (zh) 2012-11-27 2022-09-23 儿童医疗中心有限公司 用于胎儿血红蛋白再诱导的靶向bcl11a远端调控元件
AU2017252023B2 (en) * 2016-04-18 2024-05-02 Vertex Pharmaceuticals Incorporated Materials and methods for treatment of hemoglobinopathies
DK3636753T5 (da) 2017-06-08 2024-08-26 Univ Osaka Fremgangsmåde til fremstilling af DNA-redigeret eukaryotisk celle
AU2018378479B2 (en) 2017-12-05 2025-03-13 Vertex Pharmaceuticals Incorporated CRISPR-Cas9 modified CD34+ human hematopoietic stem and progenitor cells and uses thereof
WO2020184723A1 (ja) * 2019-03-14 2020-09-17 国立大学法人徳島大学 Crisprタイプi-dシステムを利用した標的配列改変技術
JP2022547105A (ja) * 2019-09-04 2022-11-10 博雅▲輯▼因(北京)生物科技有限公司 オフターゲット評価に基づく遺伝子編集治療の評価方法
KR20230150998A (ko) * 2021-03-01 2023-10-31 씨포유 가부시키가이샤 Cas3 단백질을 제조하는 방법

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JPWO2025094917A1 (https=) 2025-05-08
WO2025094917A1 (ja) 2025-05-08

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