MX2023004479A - Sistema de vector multiple y sus usos. - Google Patents
Sistema de vector multiple y sus usos.Info
- Publication number
- MX2023004479A MX2023004479A MX2023004479A MX2023004479A MX2023004479A MX 2023004479 A MX2023004479 A MX 2023004479A MX 2023004479 A MX2023004479 A MX 2023004479A MX 2023004479 A MX2023004479 A MX 2023004479A MX 2023004479 A MX2023004479 A MX 2023004479A
- Authority
- MX
- Mexico
- Prior art keywords
- vector system
- multiple vector
- constructs
- vectors
- allow
- Prior art date
Links
- 239000013598 vector Substances 0.000 title abstract 2
- 238000001415 gene therapy Methods 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 108090000623 proteins and genes Proteins 0.000 abstract 1
Classifications
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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
-
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P21/00—Drugs for disorders of the muscular or neuromuscular system
- A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P9/00—Drugs for disorders of the cardiovascular system
- A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
-
- 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
-
- 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
-
- 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
- C12N2840/00—Vectors comprising a special translation-regulating system
- C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
-
- 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
Abstract
La presente invención se refiere a constructos, vectores, células hospederas relativas y composiciones farmacéuticas que permiten una terapia genética eficaz, en particular de genes mayores a 5Kb.
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562127463P | 2015-03-03 | 2015-03-03 |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2023004479A true MX2023004479A (es) | 2023-05-04 |
Family
ID=56849204
Family Applications (2)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2017011255A MX2017011255A (es) | 2015-03-03 | 2016-03-03 | Sistema de vector múltiple y sus usos. |
MX2023004479A MX2023004479A (es) | 2015-03-03 | 2017-08-31 | Sistema de vector multiple y sus usos. |
Family Applications Before (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2017011255A MX2017011255A (es) | 2015-03-03 | 2016-03-03 | Sistema de vector múltiple y sus usos. |
Country Status (17)
Country | Link |
---|---|
US (2) | US20180327779A1 (es) |
EP (2) | EP3265571B1 (es) |
JP (2) | JP7182873B2 (es) |
KR (1) | KR102240180B1 (es) |
CN (1) | CN107466325A (es) |
AU (1) | AU2016227668B2 (es) |
BR (1) | BR112017018728A2 (es) |
CA (1) | CA2979120A1 (es) |
DK (1) | DK3265571T3 (es) |
ES (1) | ES2919880T3 (es) |
HR (1) | HRP20220776T1 (es) |
MX (2) | MX2017011255A (es) |
PL (1) | PL3265571T3 (es) |
PT (1) | PT3265571T (es) |
RS (1) | RS63416B1 (es) |
SI (1) | SI3265571T1 (es) |
WO (1) | WO2016139321A1 (es) |
Families Citing this family (16)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP3719134B1 (en) * | 2015-03-11 | 2023-08-23 | The United States of America, as represented by The Secretary, Department of Health and Human Services | Rp2 vectors for treating x-linked retinitis pigmentosa |
US11072803B2 (en) | 2015-12-22 | 2021-07-27 | Inserm (Institut National De La Sante Et De La Recherche Medicale) | Hybrid dual recombinant AAV vector systems for gene therapy |
JP2019530737A (ja) | 2016-08-23 | 2019-10-24 | アコーオス インコーポレイテッド | ヒト対象において非加齢性聴力障害を治療するための組成物および方法 |
CN110892062A (zh) * | 2017-05-05 | 2020-03-17 | 佛罗里达大学研究基金会 | 表达耳畸蛋白的组合物和方法 |
CN113056561A (zh) * | 2018-04-05 | 2021-06-29 | 牛津大学科技创新有限公司 | 用于治疗黄斑营养不良的组合物和方法 |
EP3781213A4 (en) * | 2018-04-17 | 2022-10-19 | The Trustees of the University of Pennsylvania | TRANS-SPLICE MOLECULES |
US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
EP3867387A2 (en) * | 2018-10-15 | 2021-08-25 | Fondazione Telethon | Intein proteins and uses thereof |
EP3870148A4 (en) * | 2018-10-25 | 2022-11-09 | Takeda Pharmaceutical Company Limited | AAV TRIPLE PLASMID SYSTEM |
EP3956453A4 (en) * | 2019-04-19 | 2023-02-22 | University of Massachusetts | GENE THERAPIES FOR USHER SYNDROME (USH1B) |
US20220315948A1 (en) * | 2019-04-26 | 2022-10-06 | President And Fellows Of Harvard College | Aav vectors encoding mini-pcdh15 and uses thereof |
WO2021116138A1 (en) * | 2019-12-09 | 2021-06-17 | Ucl Business Ltd | Gene therapy composition and treatment for myh7-linked cardiomyopathy |
EP4100518A2 (en) * | 2020-02-07 | 2022-12-14 | The Children's Medical Center Corporation | Large gene vectors and delivery and uses thereof |
AU2021225035A1 (en) | 2020-02-21 | 2022-10-13 | Akouos, Inc. | Compositions and methods for treating non-age-associated hearing impairment in a human subject |
CN112501209B (zh) * | 2020-12-07 | 2024-02-13 | 和元生物技术(上海)股份有限公司 | 外源基因可控表达的腺相关病毒包装方法 |
CA3218195A1 (en) * | 2021-05-07 | 2022-11-10 | Robin Ali | Abca4 genome editing |
Family Cites Families (14)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5827702A (en) | 1994-10-31 | 1998-10-27 | Genentech, Inc. | Ocular gene therapy |
GB9720465D0 (en) * | 1997-09-25 | 1997-11-26 | Oxford Biomedica Ltd | Dual-virus vectors |
US6106826A (en) | 1997-12-17 | 2000-08-22 | Wisconsin Alumni Research Foundation | Replication competent, avirulent Herpes simplex virus as a vector for neural and ocular gene therapy |
DK1071806T3 (da) | 1998-04-24 | 2004-07-19 | Univ Florida | Rekombinant adeno-associeret virus-vektor, der koder for alpha-1-antitrypsin i genterapi |
US6967018B2 (en) | 2002-01-11 | 2005-11-22 | Applied Genetic Technologies Corporation | Adiponectin gene therapy |
WO2003088916A2 (en) * | 2002-04-19 | 2003-10-30 | Georgia Tech Research Corporation | Compositions and methods for the acceleration of protein secretion dynamics |
WO2006036465A2 (en) | 2004-09-03 | 2006-04-06 | University Of Florida | Compositions and methods for treating cystic fibrosis |
JP4948845B2 (ja) * | 2006-02-08 | 2012-06-06 | 財団法人 東京都医学総合研究所 | 神経変性疾患治療用物質のスクリーニング方法 |
AU2007223427A1 (en) * | 2006-03-01 | 2007-09-13 | University Of Utah Research Foundation | Methods and compositions related to cyclic peptide synthesis |
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 |
US8236557B2 (en) * | 2008-05-28 | 2012-08-07 | University Of Missouri-Columbia | Hybrid-AAV vectors to deliver large gene expression cassette |
WO2010059763A1 (en) * | 2008-11-19 | 2010-05-27 | Amyris Biotechnologies, Inc. | Compositions and methods for the assembly of polynucleotides |
AU2010267963B2 (en) * | 2009-07-02 | 2015-09-24 | Merz Pharma Gmbh & Co. Kgaa | Neurotoxins exhibiting shortened biological activity |
ES2704677T3 (es) * | 2013-04-18 | 2019-03-19 | Fond Telethon | Suministro eficaz de genes grandes por vectores de AAV duales |
-
2016
- 2016-03-03 EP EP16710113.8A patent/EP3265571B1/en active Active
- 2016-03-03 HR HRP20220776TT patent/HRP20220776T1/hr unknown
- 2016-03-03 BR BR112017018728A patent/BR112017018728A2/pt not_active Application Discontinuation
- 2016-03-03 US US15/555,479 patent/US20180327779A1/en not_active Abandoned
- 2016-03-03 CA CA2979120A patent/CA2979120A1/en active Pending
- 2016-03-03 WO PCT/EP2016/054602 patent/WO2016139321A1/en active Application Filing
- 2016-03-03 AU AU2016227668A patent/AU2016227668B2/en active Active
- 2016-03-03 PL PL16710113.8T patent/PL3265571T3/pl unknown
- 2016-03-03 DK DK16710113.8T patent/DK3265571T3/da active
- 2016-03-03 PT PT167101138T patent/PT3265571T/pt unknown
- 2016-03-03 SI SI201631554T patent/SI3265571T1/sl unknown
- 2016-03-03 MX MX2017011255A patent/MX2017011255A/es unknown
- 2016-03-03 CN CN201680013732.1A patent/CN107466325A/zh active Pending
- 2016-03-03 RS RS20220591A patent/RS63416B1/sr unknown
- 2016-03-03 JP JP2017546224A patent/JP7182873B2/ja active Active
- 2016-03-03 EP EP22160356.6A patent/EP4089172A3/en active Pending
- 2016-03-03 KR KR1020177027977A patent/KR102240180B1/ko active IP Right Grant
- 2016-03-03 ES ES16710113T patent/ES2919880T3/es active Active
-
2017
- 2017-08-31 MX MX2023004479A patent/MX2023004479A/es unknown
-
2021
- 2021-03-15 US US17/202,098 patent/US20220002749A1/en active Pending
-
2022
- 2022-09-07 JP JP2022142232A patent/JP2022174192A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
WO2016139321A1 (en) | 2016-09-09 |
PL3265571T3 (pl) | 2022-09-05 |
BR112017018728A2 (pt) | 2018-04-17 |
DK3265571T3 (da) | 2022-06-27 |
KR102240180B1 (ko) | 2021-04-14 |
AU2016227668B2 (en) | 2019-06-27 |
CN107466325A (zh) | 2017-12-12 |
PT3265571T (pt) | 2022-06-29 |
JP2018512125A (ja) | 2018-05-17 |
MX2017011255A (es) | 2018-08-01 |
JP7182873B2 (ja) | 2022-12-05 |
EP3265571B1 (en) | 2022-04-13 |
KR20170140185A (ko) | 2017-12-20 |
EP4089172A3 (en) | 2023-03-01 |
RS63416B1 (sr) | 2022-08-31 |
EP4089172A2 (en) | 2022-11-16 |
CA2979120A1 (en) | 2016-09-09 |
AU2016227668A1 (en) | 2017-08-31 |
ES2919880T3 (es) | 2022-07-28 |
US20220002749A1 (en) | 2022-01-06 |
US20180327779A1 (en) | 2018-11-15 |
EP3265571A1 (en) | 2018-01-10 |
HRP20220776T1 (hr) | 2022-09-16 |
SI3265571T1 (sl) | 2022-10-28 |
JP2022174192A (ja) | 2022-11-22 |
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