IL284021A - An optimized acceptor splice site module for biological and biotechnological applications - Google Patents
An optimized acceptor splice site module for biological and biotechnological applicationsInfo
- Publication number
- IL284021A IL284021A IL284021A IL28402121A IL284021A IL 284021 A IL284021 A IL 284021A IL 284021 A IL284021 A IL 284021A IL 28402121 A IL28402121 A IL 28402121A IL 284021 A IL284021 A IL 284021A
- Authority
- IL
- Israel
- Prior art keywords
- biological
- unit adapted
- site unit
- receive fusion
- biotechnological uses
- Prior art date
Links
- 230000004927 fusion Effects 0.000 title 1
Classifications
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
- A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
- A61P27/02—Ophthalmic agents
-
- 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
- C12N15/10—Processes for the isolation, preparation or purification of DNA or RNA
- C12N15/102—Mutagenizing nucleic acids
-
- 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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- 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
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/33—Alteration of splicing
-
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
-
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
- C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Medicinal Chemistry (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Mycology (AREA)
- Crystallography & Structural Chemistry (AREA)
- Ophthalmology & Optometry (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP18214415 | 2018-12-20 | ||
| PCT/EP2019/086454 WO2020127831A1 (en) | 2018-12-20 | 2019-12-19 | An optimized acceptor splice site module for biological and biotechnological applications |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| IL284021A true IL284021A (en) | 2021-08-31 |
Family
ID=64755152
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| IL284021A IL284021A (en) | 2018-12-20 | 2021-06-15 | An optimized acceptor splice site module for biological and biotechnological applications |
Country Status (10)
| Country | Link |
|---|---|
| US (1) | US12502438B2 (https=) |
| EP (1) | EP3898996A1 (https=) |
| JP (2) | JP7668737B2 (https=) |
| KR (1) | KR102938671B1 (https=) |
| CN (1) | CN113474460A (https=) |
| AU (1) | AU2019406702B2 (https=) |
| CA (1) | CA3122909A1 (https=) |
| IL (1) | IL284021A (https=) |
| SG (1) | SG11202106356QA (https=) |
| WO (1) | WO2020127831A1 (https=) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SG11202106356QA (en) * | 2018-12-20 | 2021-07-29 | Vigeneron Gmbh | An optimized acceptor splice site module for biological and biotechnological applications |
| WO2020205604A1 (en) | 2019-03-29 | 2020-10-08 | Salk Institute For Biological Studies | High-efficiency reconstitution of rna molecules |
| US20230287458A1 (en) * | 2020-07-14 | 2023-09-14 | Abeona Therapeutics Inc. | Recombinant adeno-associated viral vectors for multipartite gene delivery |
| US20230374476A1 (en) * | 2021-02-05 | 2023-11-23 | University Of Massachusetts | Prime editor system for in vivo genome editing |
| WO2022220968A1 (en) * | 2021-04-15 | 2022-10-20 | U1 Bio, Inc. | High efficiency trans-splicing for replacement of targeted rna sequences in human cells |
| JP2024517939A (ja) * | 2021-05-14 | 2024-04-23 | ソーク インスティテュート フォー バイオロジカル スタディーズ | 編集タンパク質の発現のための方法および組成物 |
| CN116463380A (zh) * | 2023-03-30 | 2023-07-21 | 中国科学院深圳先进技术研究院 | 一种重组腺相关病毒载体组合物、外源基因表达系统及方法 |
Family Cites Families (16)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5478745A (en) | 1992-12-04 | 1995-12-26 | University Of Pittsburgh | Recombinant viral vector system |
| US20030077754A1 (en) * | 1995-12-15 | 2003-04-24 | Mitchell Lloyd G. | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| US20020193580A1 (en) * | 1995-12-15 | 2002-12-19 | Mitchell Lloyd G. | Methods and compositions for use in spliceosome mediated RNA trans-splicing |
| US6436707B1 (en) * | 1998-03-27 | 2002-08-20 | Lexicon Genetics Incorporated | Vectors for gene mutagenesis and gene discovery |
| JP2009000120A (ja) | 1998-08-13 | 2009-01-08 | Virxsys Corp | スプライソソーム媒介ｒｎａトランス−スプライシングにおける使用のための方法及び組成物 |
| AU780231B2 (en) | 1998-11-10 | 2005-03-10 | University Of North Carolina At Chapel Hill, The | Virus vectors and methods of making and administering the same |
| GB9906615D0 (en) | 1999-03-22 | 1999-05-19 | Oxford Biomedica Ltd | Vector |
| CA2477295A1 (en) | 2002-02-25 | 2003-09-04 | Intronn, Inc. | Trans-splicing mediated imaging of gene expression |
| US7968334B2 (en) | 2004-01-23 | 2011-06-28 | Virxsys Corporation | Expression of apoAI and variants thereof using spliceosome mediated RNA trans-splicing |
| US9303078B2 (en) | 2009-10-08 | 2016-04-05 | Association Institut De Myologie | Nucleic acid molecules and methods for exchanging exon(s) by transsplicing |
| SG2014012207A (en) | 2011-08-12 | 2014-05-29 | Virxsys Corp | Compositions and methods for inducing apoptosis |
| WO2013075008A1 (en) | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| AU2014255665B2 (en) | 2013-04-18 | 2018-08-02 | Fondazione Telethon | Effective delivery of large genes by dual AAV vectors |
| JP7007273B2 (ja) * | 2015-12-22 | 2022-01-24 | アンセルム（アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル） | 遺伝子治療用の改良された複合型二重組換えａａｖベクターシステム |
| US12043848B2 (en) | 2017-10-16 | 2024-07-23 | Vigeneron Gmbh | AAV vectors |
| SG11202106356QA (en) * | 2018-12-20 | 2021-07-29 | Vigeneron Gmbh | An optimized acceptor splice site module for biological and biotechnological applications |
-
2019
- 2019-12-19 SG SG11202106356QA patent/SG11202106356QA/en unknown
- 2019-12-19 EP EP19829184.1A patent/EP3898996A1/en active Pending
- 2019-12-19 WO PCT/EP2019/086454 patent/WO2020127831A1/en not_active Ceased
- 2019-12-19 US US17/415,643 patent/US12502438B2/en active Active
- 2019-12-19 AU AU2019406702A patent/AU2019406702B2/en active Active
- 2019-12-19 CA CA3122909A patent/CA3122909A1/en active Pending
- 2019-12-19 JP JP2021534372A patent/JP7668737B2/ja active Active
- 2019-12-19 CN CN201980092298.4A patent/CN113474460A/zh active Pending
- 2019-12-19 KR KR1020217018661A patent/KR102938671B1/ko active Active
-
2021
- 2021-06-15 IL IL284021A patent/IL284021A/en unknown
-
2024
- 2024-12-25 JP JP2024229104A patent/JP2025060848A/ja active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CN113474460A (zh) | 2021-10-01 |
| US20220160898A1 (en) | 2022-05-26 |
| JP2022515606A (ja) | 2022-02-21 |
| JP7668737B2 (ja) | 2025-04-25 |
| AU2019406702A1 (en) | 2021-06-10 |
| SG11202106356QA (en) | 2021-07-29 |
| KR20210125472A (ko) | 2021-10-18 |
| EP3898996A1 (en) | 2021-10-27 |
| AU2019406702B2 (en) | 2025-12-18 |
| KR102938671B1 (ko) | 2026-03-11 |
| CA3122909A1 (en) | 2020-06-25 |
| JP2025060848A (ja) | 2025-04-10 |
| WO2020127831A1 (en) | 2020-06-25 |
| US12502438B2 (en) | 2025-12-23 |
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