BR112022019167A2 - Inteínas split e usos das mesmas - Google Patents
Inteínas split e usos das mesmasInfo
- Publication number
- BR112022019167A2 BR112022019167A2 BR112022019167A BR112022019167A BR112022019167A2 BR 112022019167 A2 BR112022019167 A2 BR 112022019167A2 BR 112022019167 A BR112022019167 A BR 112022019167A BR 112022019167 A BR112022019167 A BR 112022019167A BR 112022019167 A2 BR112022019167 A2 BR 112022019167A2
- Authority
- BR
- Brazil
- Prior art keywords
- split inteins
- inteins
- split
- degrons
- combining
- Prior art date
Links
- 230000015556 catabolic process Effects 0.000 abstract 1
- 238000006731 degradation reaction Methods 0.000 abstract 1
- 230000001687 destabilization Effects 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 230000017730 intein-mediated protein splicing Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
Classifications
-
- 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]
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)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| 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 (1)
| Publication Number | Publication Date |
|---|---|
| BR112022019167A2 true BR112022019167A2 (pt) | 2022-11-01 |
Family
ID=70292931
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| BR112022019167A BR112022019167A2 (pt) | 2020-03-26 | 2021-03-26 | Inteínas split e usos das mesmas |
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)
| 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 |
Family Cites Families (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| SI3265571T1 (sl) * | 2015-03-03 | 2022-10-28 | Fondazione Telethon | Več vektorski sistem in njegove uporabe |
| 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 |
-
2020
- 2020-03-26 EP EP20382236.6A patent/EP3885440A1/en active Pending
-
2021
- 2021-03-26 EP EP21713447.7A patent/EP4127194A1/en active Pending
- 2021-03-26 CA CA3175696A patent/CA3175696A1/en active Pending
- 2021-03-26 KR KR1020227037261A patent/KR20220160052A/ko active Pending
- 2021-03-26 JP JP2022558063A patent/JP2023518596A/ja active Pending
- 2021-03-26 WO PCT/EP2021/058016 patent/WO2021191447A1/en not_active Ceased
- 2021-03-26 CN CN202180038835.4A patent/CN115698307A/zh active Pending
- 2021-03-26 US US17/914,212 patent/US20230116688A1/en active Pending
- 2021-03-26 BR BR112022019167A patent/BR112022019167A2/pt unknown
- 2021-03-26 AU AU2021241899A patent/AU2021241899A1/en active Pending
- 2021-03-26 IL IL296712A patent/IL296712A/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| US20230116688A1 (en) | 2023-04-13 |
| CA3175696A1 (en) | 2021-09-30 |
| JP2023518596A (ja) | 2023-05-02 |
| 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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