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 (https=
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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)
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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)
  • Chemical Kinetics & Catalysis (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Neurology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Epidemiology (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes 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 2020-12-16
EP20214691.6 2020-12-16
EP21209721 2021-11-22
EP21209721.6 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 (https=) 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

Country Status (7)

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

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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

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)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8048999B2 (en) 2005-12-13 2011-11-01 Kyoto University Nuclear reprogramming factor
JP5813321B2 (ja) 2007-03-23 2015-11-17 ウィスコンシン アラムニ リサーチ ファンデーション 体細胞の再プログラム化
US20200095579A1 (en) * 2017-02-22 2020-03-26 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
US20220235379A1 (en) * 2019-06-11 2022-07-28 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
EP4263818A1 (en) 2023-10-25
KR20230125806A (ko) 2023-08-29
AU2021399929A1 (en) 2023-07-06
CA3202411A1 (en) 2022-06-23
US20250269057A1 (en) 2025-08-28
WO2022129430A1 (en) 2022-06-23

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