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 (https=
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)
  • 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)
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
EP20382236.6A 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 (2)

Publication Number Publication Date
JP2023518596A true JP2023518596A (ja) 2023-05-02
JP2023518596A5 JP2023518596A5 (https=) 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 (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=)

Families Citing this family (13)

* 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
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
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 (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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US5100792A (en) 1984-11-13 1992-03-31 Cornell Research Foundation, Inc. Method for transporting substances into living cells and tissues
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

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., 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
US20230116688A1 (en) 2023-04-13
CA3175696A1 (en) 2021-09-30
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
CN115698307A (zh) 2023-02-03
WO2021191447A1 (en) 2021-09-30
EP3885440A1 (en) 2021-09-29

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