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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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
    • 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]

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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)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Preparation Of Compounds By Using 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 (https=)
EP (2) EP3885440A1 (https=)
JP (1) JP2023518596A (https=)
KR (1) KR20220160052A (https=)
CN (1) CN115698307A (https=)
AU (1) AU2021241899A1 (https=)
BR (1) BR112022019167A2 (https=)
CA (1) CA3175696A1 (https=)
IL (1) IL296712A (https=)
WO (1) WO2021191447A1 (https=)

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

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3847254A4 (en) 2018-09-07 2022-08-10 Beam Therapeutics Inc. COMPOSITIONS AND METHODS FOR DELIVERING A NUCLEOBASE EDITING SYSTEM
JP7579844B2 (ja) * 2019-08-28 2024-11-08 ザ・トラスティーズ・オブ・プリンストン・ユニバーシティ 非定型スプリットインテインおよびそれらの使用
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
US20250319126A1 (en) * 2021-10-29 2025-10-16 Shanghai Sinobay Biotechnology Co., Ltd. Condition-controlled spliceable chimeric antigen receptor molecule and application thereof
JP2025530726A (ja) * 2022-05-17 2025-09-17 復旦大学附属眼耳鼻喉科医院 聴力障害を治療するためのデュアルベクターシステム及びその使用
JP2026504390A (ja) * 2023-02-01 2026-02-05 アレン インスティテュート インテインを介した電位依存性ナトリウムチャネルの機能の再構築
WO2025038955A1 (en) * 2023-08-16 2025-02-20 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

Citations (1)

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Publication number Priority date Publication date Assignee Title
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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US4945050A (en) 1984-11-13 1990-07-31 Cornell Research Foundation, Inc. Method for transporting substances into living cells and tissues and apparatus therefor
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.
FR2727679B1 (fr) 1994-12-05 1997-01-03 Rhone Poulenc Rorer Sa Nouveaux agents de transfection et leurs applications pharmaceutiques
FR2730637B1 (fr) 1995-02-17 1997-03-28 Rhone Poulenc Rorer Sa Composition pharmaceutique contenant des acides nucleiques, et ses utilisations
WO2007127428A2 (en) 2006-04-28 2007-11-08 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
EP3312279B1 (en) * 2015-06-19 2020-10-14 Riken pH-RESPONSIVE PROTEOLYSIS PROBE
PT3408292T (pt) * 2016-01-29 2023-07-19 Univ Princeton Inteínas divididas com excecional atividade de splicing
IL282362B2 (en) * 2018-10-15 2025-10-01 Fond Telethon Ets Intein proteins and their uses

Patent 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

Non-Patent Citations (3)

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

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