JP7867703B2 - スプリットインテイン及びその使用 - Google Patents

スプリットインテイン及びその使用

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Publication number
JP7867703B2
JP7867703B2 JP2022558063A JP2022558063A JP7867703B2 JP 7867703 B2 JP7867703 B2 JP 7867703B2 JP 2022558063 A JP2022558063 A JP 2022558063A JP 2022558063 A JP2022558063 A JP 2022558063A JP 7867703 B2 JP7867703 B2 JP 7867703B2
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JP
Japan
Prior art keywords
fragment
protein
split
intein
degron
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JP2022558063A
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English (en)
Japanese (ja)
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JP2023518596A5 (enExample
JP2023518596A (ja
Inventor
ドミンゲス シルビア フルートス
ヴィダル ジェラルド カエレス
ビルバオ アナベル オテロ
ペレロ ミケル ヴィラ
Original Assignee
スプライスバイオ エセ．エレ．
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Publication of JP2023518596A5 publication Critical patent/JP2023518596A5/ja
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Classifications

    • 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

Landscapes

  • 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)
JP2022558063A 2020-03-26 2021-03-26 スプリットインテイン及びその使用 Active JP7867703B2 (ja)

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2026080696A Division JP2026130000A (ja) 2020-03-26 2026-05-13 スプリットインテイン及びその使用

Publications (3)

Publication Number Publication Date
JP2023518596A JP2023518596A (ja) 2023-05-02
JP2023518596A5 JP2023518596A5 (enExample) 2024-04-05
JP7867703B2 true JP7867703B2 (ja) 2026-06-01

Family

ID=70292931

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022558063A Active JP7867703B2 (ja) 2020-03-26 2021-03-26 スプリットインテイン及びその使用

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)

Families Citing this family (15)

* Cited by examiner, † Cited by third party
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
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
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 (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018512125A (ja) 2015-03-03 2018-05-17 フォンダッツィオーネ・テレソン 多重ベクターシステム及びその使用
JP2019506163A (ja) 2016-01-29 2019-03-07 ザ・トラスティーズ・オブ・プリンストン・ユニバーシティＴｈｅ Ｔｒｕｓｔｅｅｓ Ｏｆ Ｐｒｉｎｃｅｔｏｎ Ｕｎｉｖｅｒｓｉｔｙ 例外的なスプライシング活性を有するスプリットインテイン
JP2022512718A (ja) 2018-10-15 2022-02-07 フォンダッツィオーネ・テレソン インテインタンパク質及びその使用

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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
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.
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
US8298818B2 (en) 2006-04-28 2012-10-30 University Of Florida Research Foundation, Inc. Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter
SG11201710569YA (en) * 2015-06-19 2018-01-30 Riken pH-RESPONSIVE PROTEOLYSIS PROBE

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2018512125A (ja) 2015-03-03 2018-05-17 フォンダッツィオーネ・テレソン 多重ベクターシステム及びその使用
JP2019506163A (ja) 2016-01-29 2019-03-07 ザ・トラスティーズ・オブ・プリンストン・ユニバーシティＴｈｅ Ｔｒｕｓｔｅｅｓ Ｏｆ Ｐｒｉｎｃｅｔｏｎ Ｕｎｉｖｅｒｓｉｔｙ 例外的なスプライシング活性を有するスプリットインテイン
JP2022512718A (ja) 2018-10-15 2022-02-07 フォンダッツィオーネ・テレソン インテインタンパク質及びその使用

Non-Patent Citations (3)

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Title
Proceedings of the National Academy of Sciences, U.S.A.，2017年，Vol.114, No.32，pp.8538-8543，doi:10.1073.pnas.1701083114
Protein Science，2018年，Vol.27，pp.614-619，doi:10.1002/pro.3357
Science Translational Medicine，2019年，Vol.11，aav4523，doi:10.1126/scitranslmed.aav4523

Also Published As

Publication number Publication date
EP4127194A1 (en) 2023-02-08
EP3885440A1 (en) 2021-09-29
WO2021191447A1 (en) 2021-09-30
CA3175696A1 (en) 2021-09-30
CN115698307A (zh) 2023-02-03
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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