JP2023518596A - 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 Download PDF

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JP2023518596A
JP2023518596A JP2022558063A JP2022558063A JP2023518596A JP 2023518596 A JP2023518596 A JP 2023518596A JP 2022558063 A JP2022558063 A JP 2022558063A JP 2022558063 A JP2022558063 A JP 2022558063A JP 2023518596 A JP2023518596 A JP 2023518596A
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fragment
intein
degron
protein
sope
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JP2023518596A5 (enExample
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
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スプライスバイオ エセ．エレ．
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Publication of JP2023518596A publication Critical patent/JP2023518596A/ja
Publication of JP2023518596A5 publication Critical patent/JP2023518596A5/ja
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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
    • 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
    • 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
    • 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)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Plant Pathology (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)
JP2022558063A 2020-03-26 2021-03-26 スプリットインテイン及びその使用 Pending JP2023518596A (ja)

Applications Claiming Priority (3)

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

Publications (2)

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

Family

ID=70292931

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2022558063A Pending JP2023518596A (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) JP2023518596A (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 (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2019336245B2 (en) 2018-09-07 2026-02-26 Beam Therapeutics Inc. Compositions and methods for delivering a nucleobase editing system
CA3152679A1 (en) * 2019-08-28 2021-03-04 The Trustees Of Princeton University Atypical split inteins and uses thereof
CN116096905A (zh) 2020-02-21 2023-05-09 阿库斯股份有限公司 用于治疗人受试者的非年龄相关性听力损伤的组合物和方法
EP4424714A4 (en) * 2021-10-29 2025-10-15 Shanghai Sinobay Biotechnology Co Ltd Condition-Controlled Splicable Chimeric Antigen Receptor Molecule and Its Use
EP4527933A4 (en) * 2022-05-17 2025-12-24 Eye & Ent Hospital Of Fudan Univ DUAL-SUPPORT SYSTEM FOR THE TREATMENT OF HEARING LOSS, AND ITS USE
KR20250141200A (ko) * 2023-02-01 2025-09-26 알렌 인스티튜트 전압-게이팅 나트륨 채널 기능의 인테인 매개 재구성
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
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
AU2024338787A1 (en) 2023-09-04 2026-03-19 Splicebio, Sl Use of split intein for the treatment of scn1a associated disease
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 (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

Family Cites Families (11)

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Publication number Priority date Publication date Assignee Title
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
US5100792A (en) 1984-11-13 1992-03-31 Cornell Research Foundation, Inc. Method for transporting substances into living cells and tissues
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
US11203621B2 (en) * 2015-06-19 2021-12-21 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)

* Cited by examiner, † Cited by third party
Title
PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES, U.S.A., vol. 114, no. 32, JPN6025006933, 2017, pages 8538 - 8543, ISSN: 0005717404 *
PROTEIN SCIENCE, vol. 27, JPN6025006932, 2018, pages 614 - 619, ISSN: 0005717406 *
SCIENCE TRANSLATIONAL MEDICINE, vol. 11, JPN6025006931, 2019, pages 4523, ISSN: 0005717405 *

Also Published As

Publication number Publication date
IL296712A (en) 2022-11-01
CN115698307A (zh) 2023-02-03
BR112022019167A2 (pt) 2022-11-01
US20230116688A1 (en) 2023-04-13
AU2021241899A1 (en) 2022-10-06
KR20220160052A (ko) 2022-12-05
EP4127194A1 (en) 2023-02-08
WO2021191447A1 (en) 2021-09-30
EP3885440A1 (en) 2021-09-29
CA3175696A1 (en) 2021-09-30

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