JP2023553701A - 先天性筋ジストロフィーの処置のための治療用ｌａｍａ２ペイロード - Google Patents

先天性筋ジストロフィーの処置のための治療用ｌａｍａ２ペイロード Download PDF

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JP2023553701A
JP2023553701A JP2023536885A JP2023536885A JP2023553701A JP 2023553701 A JP2023553701 A JP 2023553701A JP 2023536885 A JP2023536885 A JP 2023536885A JP 2023536885 A JP2023536885 A JP 2023536885A JP 2023553701 A JP2023553701 A JP 2023553701A
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protein
seq
nucleic acid
transposase
lama2
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JP2023553701A5 (enExample
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カルゴル，マルク グエル
ガルシア，アベンシア サンチェス－メジアス
マスミトジャ，マリア パラレス
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ウニベルシタット ポンペウ ファブラ
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/12—Transferases (2.) transferring phosphorus containing groups, e.g. kinases (2.7)
    • C12N9/1241—Nucleotidyltransferases (2.7.7)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/80—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor
    • C07K2319/81—Fusion polypeptide containing a DNA binding domain, e.g. Lacl or Tet-repressor containing a Zn-finger domain for DNA binding

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  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
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  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Cell Biology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Mycology (AREA)
  • Toxicology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Epidemiology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Enzymes And Modification Thereof (AREA)
JP2023536885A 2020-12-16 2021-12-16 先天性筋ジストロフィーの処置のための治療用ｌａｍａ２ペイロード Pending JP2023553701A (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EP20214691.6 2020-12-16
EP20214691 2020-12-16
EP21209721.6 2021-11-22
EP21209721 2021-11-22
PCT/EP2021/086333 WO2022129430A1 (en) 2020-12-16 2021-12-16 Therapeutic lama2 payload for treatment of congenital muscular dystrophy

Publications (2)

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JP2023553701A true JP2023553701A (ja) 2023-12-25
JP2023553701A5 JP2023553701A5 (enExample) 2024-12-23

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JP2023536885A Pending JP2023553701A (ja) 2020-12-16 2021-12-16 先天性筋ジストロフィーの処置のための治療用ｌａｍａ２ペイロード

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US (1) US20250269057A1 (enExample)
EP (1) EP4263818A1 (enExample)
JP (1) JP2023553701A (enExample)
KR (1) KR20230125806A (enExample)
AU (1) AU2021399929A1 (enExample)
CA (1) CA3202411A1 (enExample)
WO (1) WO2022129430A1 (enExample)

Families Citing this family (2)

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Publication number Priority date Publication date Assignee Title
CN118185901B (zh) * 2022-12-12 2025-03-21 北京精缮生物科技有限责任公司 一种PBase蛋白、融合蛋白、核酸和基因整合系统及应用
WO2026087488A1 (en) * 2024-10-21 2026-04-30 Integra Therapeutics Gene editing tools and compositions for cell engineering

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018513681A (ja) * 2015-03-31 2018-05-31 エクセリゲン サイエンティフィック, インコーポレイテッドExeligen Scientific, Inc. 細胞または生物のゲノムへのＤＮＡ配列の標的化組み込みのためのＣａｓ９レトロウイルスインテグラーゼおよびＣａｓ９レコンビナーゼ系
WO2018175872A1 (en) * 2017-03-24 2018-09-27 President And Fellows Of Harvard College Methods of genome engineering by nuclease-transposase fusion proteins

Family Cites Families (4)

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Publication number Priority date Publication date Assignee Title
CN101864392B (zh) 2005-12-13 2016-03-23 国立大学法人京都大学 核重新编程因子
EP3399025B1 (en) 2007-03-23 2025-05-07 Wisconsin Alumni Research Foundation Somatic cell reprogramming
EP3585896A1 (en) * 2017-02-22 2020-01-01 CRISPR Therapeutics AG Materials and methods for treatment of merosin-deficient cogenital muscular dystrophy (mdcmd) and other laminin, alpha 2 (lama2) gene related conditions or disorders
AU2020290790B2 (en) * 2019-06-11 2026-02-19 Universitat Pompeu Fabra Targeted gene editing constructs and methods of using the same

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018513681A (ja) * 2015-03-31 2018-05-31 エクセリゲン サイエンティフィック, インコーポレイテッドExeligen Scientific, Inc. 細胞または生物のゲノムへのＤＮＡ配列の標的化組み込みのためのＣａｓ９レトロウイルスインテグラーゼおよびＣａｓ９レコンビナーゼ系
WO2018175872A1 (en) * 2017-03-24 2018-09-27 President And Fellows Of Harvard College Methods of genome engineering by nuclease-transposase fusion proteins

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
SHUFENG MA ET AL.: ""Enhancing site-specific DNA integration by a Cas9 nuclease fused with a DNA donor-binding domain"", NUCLEIC ACIDS RESEARCH, vol. 48, no. 18, JPN6025044709, 28 September 2020 (2020-09-28), pages 10590 - 10601, ISSN: 0005723056 *

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Publication number Publication date
US20250269057A1 (en) 2025-08-28
AU2021399929A1 (en) 2023-07-06
EP4263818A1 (en) 2023-10-25
CA3202411A1 (en) 2022-06-23
KR20230125806A (ko) 2023-08-29
WO2022129430A1 (en) 2022-06-23

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