CN115698307A - 断裂内含肽及其用途 - Google Patents

断裂内含肽及其用途 Download PDF

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Publication number
CN115698307A
CN115698307A CN202180038835.4A CN202180038835A CN115698307A CN 115698307 A CN115698307 A CN 115698307A CN 202180038835 A CN202180038835 A CN 202180038835A CN 115698307 A CN115698307 A CN 115698307A
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China
Prior art keywords
fragment
intein
leu
protein
degron
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Pending
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CN202180038835.4A
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English (en)
Chinese (zh)
Inventor
西尔维娅·弗鲁托·多明格斯
杰拉德·凯莱斯·维达尔
安娜贝尔·奥特罗·毕尔巴鄂
米克尔·维拉·佩雷洛
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Mobile Biology Co ltd
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Mobile Biology Co ltd
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Publication of CN115698307A publication Critical patent/CN115698307A/zh
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    • 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
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/60—Fusion polypeptide containing spectroscopic/fluorescent detection, e.g. green fluorescent protein [GFP]
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/90—Fusion polypeptide containing a motif for post-translational modification
    • C07K2319/92—Fusion polypeptide containing a motif for post-translational modification containing an intein ("protein splicing")domain
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2800/00—Nucleic acids vectors
    • C12N2800/40—Systems of functionally co-operating vectors

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Zoology (AREA)
  • Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202180038835.4A 2020-03-26 2021-03-26 断裂内含肽及其用途 Pending CN115698307A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP20382236.6A EP3885440A1 (en) 2020-03-26 2020-03-26 Split inteins and their uses
EP20382236.6 2020-03-26
PCT/EP2021/058016 WO2021191447A1 (en) 2020-03-26 2021-03-26 Split inteins and their uses

Publications (1)

Publication Number Publication Date
CN115698307A true CN115698307A (zh) 2023-02-03

Family

ID=70292931

Family Applications (1)

Application Number Title Priority Date Filing Date
CN202180038835.4A Pending CN115698307A (zh) 2020-03-26 2021-03-26 断裂内含肽及其用途

Country Status (10)

Country Link
US (1) US20230116688A1 (enExample)
EP (2) EP3885440A1 (enExample)
JP (1) JP7867703B2 (enExample)
KR (1) KR20220160052A (enExample)
CN (1) CN115698307A (enExample)
AU (1) AU2021241899A1 (enExample)
BR (1) BR112022019167A2 (enExample)
CA (1) CA3175696A1 (enExample)
IL (1) IL296712A (enExample)
WO (1) WO2021191447A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024251077A1 (zh) 2023-06-07 2024-12-12 北京中因科技有限公司 表达盒组合及其应用

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Publication number Priority date Publication date Assignee Title
IL325122A (en) 2018-02-22 2026-02-01 Akouos Inc Compositions and methods for treating non-age-related hearing impairment in humans
KR20210055733A (ko) 2018-09-07 2021-05-17 빔 테라퓨틱스, 인크. 핵염기 편집 시스템을 전달하기 위한 조성물 및 방법
WO2021040703A1 (en) * 2019-08-28 2021-03-04 The Trustees Of Princeton University Atypical split inteins and uses thereof
CA3168055A1 (en) 2020-02-21 2021-08-26 Emmanuel John Simons Compositions and methods for treating non-age-associated hearing impairment in a human subject
US20250319126A1 (en) * 2021-10-29 2025-10-16 Shanghai Sinobay Biotechnology Co., Ltd. Condition-controlled spliceable chimeric antigen receptor molecule and application thereof
CN117106824B (zh) * 2022-05-17 2025-09-02 上海佑音医药生物科技有限公司 一种治疗听力损伤的双载体系统及其应用
KR20250141200A (ko) * 2023-02-01 2025-09-26 알렌 인스티튜트 전압-게이팅 나트륨 채널 기능의 인테인 매개 재구성
EP4762183A1 (en) * 2023-08-16 2026-06-24 Seattle Children's Hospital d/b/a Seattle Children's Research Institute Systems and methods for improving safety of split intein aav mediated gene therapy
CN121909039A (zh) 2023-09-04 2026-04-21 斯普莱斯生物有限责任公司 分裂内含肽用于治疗scn1a相关疾病的用途
WO2025153705A1 (en) 2024-01-18 2025-07-24 Splicebio, Sl Use of split intein for the treatment of myo7a-associated disease
WO2025193938A1 (en) * 2024-03-13 2025-09-18 Oregon Health & Science University Delivery of type iv collagen alpha chain using a split intein dual aav vector
WO2026013288A1 (en) 2024-07-12 2026-01-15 Splicebio, Sl Split intein for use in the treatment of cep290-associated disease
WO2026057767A1 (en) 2024-09-13 2026-03-19 Splicebio, Sl Use of split intein for the treatment of abca3-associated disease
WO2026109723A1 (en) * 2024-11-22 2026-05-28 Katholieke Universiteit Leuven Efficient intein splicing in dual aav gene delivery

Citations (1)

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CN107466325A (zh) * 2015-03-03 2017-12-12 泰莱托恩基金会 多重载体系统及其应用

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US5100792A (en) 1984-11-13 1992-03-31 Cornell Research Foundation, Inc. Method for transporting substances into living cells and tissues
US5036006A (en) 1984-11-13 1991-07-30 Cornell Research Foundation, Inc. Method for transporting substances into living cells and tissues and apparatus therefor
US5703055A (en) 1989-03-21 1997-12-30 Wisconsin Alumni Research Foundation Generation of antibodies through lipid mediated DNA delivery
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
FR2714830B1 (fr) 1994-01-10 1996-03-22 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
FR2715847B1 (fr) 1994-02-08 1996-04-12 Rhone Poulenc Rorer Sa Composition contenant des acides nucléiques, préparation et utilisations.
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WO2017132580A2 (en) * 2016-01-29 2017-08-03 The Trustees Of Princeton University Split inteins with exceptional splicing activity
CA3116606A1 (en) 2018-10-15 2020-04-23 Fondazione Telethon Intein proteins and uses thereof

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DENG PAN, ET AL.: "An intein-mediated modulation of protein stability system and its application to study human cytomegalovirus essential gene function", SCIENTIFIC REPORTS, vol. 6, 18 May 2016 (2016-05-18), pages 1 - 11 *
JOSEF A GRAMESPACHER, ET AL.: "Improved protein splicing using embedded split inteins", PROTEIN SOCIETY, vol. 27, no. 3, 17 January 2018 (2018-01-17), pages 614 - 619, XP055574553, DOI: 10.1002/pro.3357 *
PATRIZIA TORNABENE, ET AL.: "Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina", SCI TRANSL MED, vol. 11, no. 492, 15 May 2019 (2019-05-15), pages 1 - 30 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024251077A1 (zh) 2023-06-07 2024-12-12 北京中因科技有限公司 表达盒组合及其应用
EP4726045A1 (en) 2023-06-07 2026-04-15 Peking University Third Hospital (The Third Clinical Medical School of Peking University) Expression cassette combination and use thereof

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Publication number Publication date
EP4127194A1 (en) 2023-02-08
EP3885440A1 (en) 2021-09-29
JP7867703B2 (ja) 2026-06-01
WO2021191447A1 (en) 2021-09-30
CA3175696A1 (en) 2021-09-30
IL296712A (en) 2022-11-01
BR112022019167A2 (pt) 2022-11-01
KR20220160052A (ko) 2022-12-05
AU2021241899A1 (en) 2022-10-06
JP2023518596A (ja) 2023-05-02
US20230116688A1 (en) 2023-04-13

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