HK1143326A1 - Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma /- - Google Patents
Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma /-Info
- Publication number
- HK1143326A1 HK1143326A1 HK10109944.6A HK10109944A HK1143326A1 HK 1143326 A1 HK1143326 A1 HK 1143326A1 HK 10109944 A HK10109944 A HK 10109944A HK 1143326 A1 HK1143326 A1 HK 1143326A1
- Authority
- HK
- Hong Kong
- Prior art keywords
- sarcolemma
- micro
- dystrophin genes
- synthetic mini
- restore nnos
- Prior art date
Links
Classifications
-
- 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
- C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
- C07K14/4707—Muscular dystrophy
- C07K14/4708—Duchenne dystrophy
-
- 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
- A61P25/00—Drugs for disorders of the nervous system
-
- 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
- A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
-
- 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
- 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
- C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
- C12N15/90—Stable introduction of foreign DNA into chromosome
- C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
- C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
-
- 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
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Biochemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Public Health (AREA)
- General Chemical & Material Sciences (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Physics & Mathematics (AREA)
- Neurology (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Cardiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Toxicology (AREA)
- Epidemiology (AREA)
- Virology (AREA)
- Mycology (AREA)
- Cell Biology (AREA)
- Heart & Thoracic Surgery (AREA)
- Neurosurgery (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US88112907P | 2007-01-18 | 2007-01-18 | |
| US99932107P | 2007-10-16 | 2007-10-16 | |
| PCT/US2008/000717 WO2008088895A2 (en) | 2007-01-18 | 2008-01-18 | Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| HK1143326A1 true HK1143326A1 (en) | 2010-12-31 |
Family
ID=39636602
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| HK10109944.6A HK1143326A1 (en) | 2007-01-18 | 2010-10-20 | Synthetic mini/micro-dystrophin genes to restore nnos to the sarcolemma /- |
Country Status (6)
| Country | Link |
|---|---|
| US (1) | US7892824B2 (xx) |
| EP (1) | EP2125006B1 (xx) |
| JP (1) | JP5575486B2 (xx) |
| CN (1) | CN101711164B (xx) |
| HK (1) | HK1143326A1 (xx) |
| WO (1) | WO2008088895A2 (xx) |
Families Citing this family (60)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1855694B1 (en) | 2005-02-09 | 2020-12-02 | Sarepta Therapeutics, Inc. | Antisense composition for treating muscle atrophy |
| US8236557B2 (en) * | 2008-05-28 | 2012-08-07 | University Of Missouri-Columbia | Hybrid-AAV vectors to deliver large gene expression cassette |
| US8889641B2 (en) | 2009-02-11 | 2014-11-18 | The University Of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| WO2011088081A1 (en) | 2010-01-12 | 2011-07-21 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
| WO2011097456A2 (en) * | 2010-02-05 | 2011-08-11 | The University Of North Carolina At Chapel Hill | Compositions and methods for enhanced parvovirus transduction |
| WO2012109570A1 (en) | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| WO2013075008A1 (en) | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Aav dual vector systems for gene therapy |
| CN103405774A (zh) * | 2012-10-24 | 2013-11-27 | 尹海芳 | 一种新型药物靶向运输辅助剂 |
| WO2014081855A1 (en) * | 2012-11-20 | 2014-05-30 | Universite De Montreal | Methods and compositions for muscular dystrophies |
| US9624282B2 (en) * | 2012-11-26 | 2017-04-18 | The Curators Of The University Of Missouri | Microdystrophin peptides and methods for treating muscular dystrophy using the same |
| US9447433B2 (en) | 2013-03-15 | 2016-09-20 | The University Of North Carolina At Chapel Hill | Synthetic adeno-associated virus inverted terminal repeats |
| WO2014144229A1 (en) | 2013-03-15 | 2014-09-18 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding aav vectors |
| JP6938377B2 (ja) | 2015-01-14 | 2021-09-22 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 標的化遺伝子移入のための方法および組成物 |
| WO2016115543A2 (en) | 2015-01-16 | 2016-07-21 | University Of Washington | Novel micro-dystrophins and related methods of use |
| WO2016134300A1 (en) | 2015-02-20 | 2016-08-25 | Board Of Regents, The University Of Texas System | Methods and compositions for attenuated chlamydia as vaccine and vector |
| GB201507842D0 (en) * | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
| EP3831842A1 (en) | 2015-09-28 | 2021-06-09 | The University of North Carolina at Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| EP3359668A4 (en) | 2015-10-09 | 2019-06-05 | Sarepta Therapeutics, Inc. | COMPOSITIONS AND METHODS OF TREATING DUCHENNE MUSCLE DYSTROPHY AND ASSOCIATED ILLNESSES THEREOF |
| CN117384964A (zh) | 2016-04-15 | 2024-01-12 | 全国儿童医院研究所 | 腺相关病毒载体递送β-肌聚糖和微RNA-29以及肌营养不良症的治疗 |
| MA45477A (fr) | 2016-04-15 | 2019-02-20 | Res Inst Nationwide Childrens Hospital | Administration à vecteurs de virus adéno-associé de microarn-29 et micro-dystrophine pour traiter la dystrophie musculaire |
| CA2971303A1 (en) * | 2016-06-21 | 2017-12-21 | Bamboo Therapeutics, Inc. | Optimized mini-dystrophin genes and expression cassettes and their use |
| US11202840B2 (en) | 2016-06-21 | 2021-12-21 | The Curators Of The University Of Missouri | Modified dystrophin proteins |
| US11584780B2 (en) | 2016-07-26 | 2023-02-21 | Biomarin Pharmaceutical Inc. | Adeno-associated virus capsid proteins |
| AU2017313844B2 (en) | 2016-08-19 | 2023-10-12 | University Of Florida Research Foundation, Incorporated | Methods and compositions for treating conditions using recombinant self-complementary adeno-associated virus |
| CN110225923A (zh) | 2016-12-07 | 2019-09-10 | 佛罗里达大学研究基金会有限公司 | IL-1Ra cDNA |
| EP3592848A1 (en) | 2017-03-10 | 2020-01-15 | Genethon | Treatment of glycogen storage disease iii |
| CN117801075A (zh) | 2017-03-15 | 2024-04-02 | 北卡罗来纳-查佩尔山大学 | 多倍体腺相关病毒载体及其制备和使用方法 |
| WO2019078916A1 (en) | 2017-10-18 | 2019-04-25 | Research Institute At Nationwide Children's Hospital | ADENO-ASSOCIATED VIRUS VECTOR ADMINISTRATION OF MUSCLE-SPECIFIC MICRO-DYSTROPHINE TO TREAT MUSCLE DYSTROPHY |
| BR112020020341A2 (pt) | 2018-04-03 | 2021-01-05 | Stridebio, Inc. | Vetores de vírus direcionados a tecidos oftálmicos |
| EP3774854A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| US20210371471A1 (en) | 2018-04-03 | 2021-12-02 | Stridebio, Inc. | Antibody-evading virus vectors |
| US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| KR20210008501A (ko) | 2018-05-09 | 2021-01-22 | 바이오마린 파머수티컬 인크. | 페닐케톤뇨증을 치료하는 방법 |
| TW202005978A (zh) | 2018-05-14 | 2020-02-01 | 美商拜奧馬林製藥公司 | 新穎肝靶向腺相關病毒載體 |
| US10758629B2 (en) * | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| CA3103485A1 (en) * | 2018-06-18 | 2019-12-26 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| EP3810782A2 (en) | 2018-06-22 | 2021-04-28 | Asklepios Biopharmaceutical, Inc. | Vectors for gene delivery that persist within cells |
| TW202102525A (zh) | 2019-03-21 | 2021-01-16 | 美商史崔德生物公司 | 重組腺相關病毒載體 |
| US20220204574A1 (en) * | 2019-03-25 | 2022-06-30 | Genethon | Production of large-sized quasidystrophins using overlapping aav vectors |
| CN113767110A (zh) | 2019-04-26 | 2021-12-07 | 北卡罗来纳-查佩尔山大学 | 用于双重聚糖结合aav2.5载体的方法和组合物 |
| CN114207120A (zh) | 2019-05-30 | 2022-03-18 | 坚固生物科技公司 | 重组疱疹病毒载体 |
| AU2020311399A1 (en) * | 2019-07-10 | 2022-02-17 | University Of Washington | Non-viral immuno-targeting |
| MX2022004352A (es) | 2019-10-17 | 2022-07-19 | Stridebio Inc | Vectores virales adenoasociados para el tratamiento de la enfermedad de niemann-pick tipo c. |
| JP2023500884A (ja) | 2019-11-06 | 2023-01-11 | アソシエーション・アンスティトゥート・ドゥ・マイオロジー | 筋疾患のための併用療法 |
| TW202134260A (zh) * | 2019-11-28 | 2021-09-16 | 美商銳進科斯生物股份有限公司 | 微小肌縮蛋白基因療法之構築體及其用途 |
| IL297753A (en) * | 2020-04-29 | 2022-12-01 | Bristol Myers Squibb Co | Minimized dystrophins with spectrin fusion domains and uses thereof |
| WO2022076750A2 (en) | 2020-10-07 | 2022-04-14 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns or muscle delivery |
| WO2022093769A1 (en) | 2020-10-28 | 2022-05-05 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding aav2.5 vector |
| EP4010356A4 (en) | 2020-10-28 | 2023-01-04 | The University of North Carolina at Chapel Hill | METHODS AND COMPOSITIONS FOR GLYCAN DOUBLE BONDING OF VAA2.5 VECTORS |
| TW202242124A (zh) | 2021-01-14 | 2022-11-01 | 美商史崔德生物公司 | 靶向ｔ細胞之ａａｖ載體 |
| EP4320139A1 (en) | 2021-04-05 | 2024-02-14 | Solid Biosciences Inc. | Recombinant herpesvirales vector |
| WO2022221529A1 (en) | 2021-04-16 | 2022-10-20 | Asklepios Biopharmaceutical, Inc. | Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response |
| IL307902A (en) * | 2021-04-26 | 2023-12-01 | Regenxbio Inc | Administering microdystrophin gene therapy to treat dystrophinopathy |
| WO2023056311A1 (en) * | 2021-09-28 | 2023-04-06 | University Of Florida Research Foundation, Incorporated | Gene therapy for duchenne muscular dystrophy |
| WO2023060269A1 (en) | 2021-10-07 | 2023-04-13 | Regenxbio Inc. | Recombinant adeno-associated viruses for targeted delivery |
| WO2023135316A1 (en) * | 2022-01-17 | 2023-07-20 | Dinaqor Ag | Gene therapy composition and treatment for dystrophin-related cardiomyopathy |
| EP4215614A1 (en) | 2022-01-24 | 2023-07-26 | Dynacure | Combination therapy for dystrophin-related diseases |
| TW202342759A (zh) | 2022-02-04 | 2023-11-01 | 美商史崔德生物公司 | 重組腺相關病毒載體及其使用方法 |
| WO2023212193A1 (en) * | 2022-04-27 | 2023-11-02 | The Curators Of The University Of Missouri | Micro-dystrophin for heart protection |
Family Cites Families (2)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2001261063A1 (en) * | 2000-04-28 | 2001-11-12 | Xiao Xiao | Dna sequences encoding dystrophin minigenes and methods of use thereof |
| EP1366160B1 (en) * | 2000-10-06 | 2008-07-09 | The Regents Of The University Of Michigan | Mini-dystrophin nucleic acid and peptide sequences |
-
2008
- 2008-01-18 JP JP2009546436A patent/JP5575486B2/ja active Active
- 2008-01-18 CN CN200880002602.3A patent/CN101711164B/zh active Active
- 2008-01-18 US US12/009,537 patent/US7892824B2/en active Active
- 2008-01-18 WO PCT/US2008/000717 patent/WO2008088895A2/en active Application Filing
- 2008-01-18 EP EP08724643.5A patent/EP2125006B1/en active Active
-
2010
- 2010-10-20 HK HK10109944.6A patent/HK1143326A1/xx unknown
Also Published As
| Publication number | Publication date |
|---|---|
| JP2010516252A (ja) | 2010-05-20 |
| JP5575486B2 (ja) | 2014-08-20 |
| EP2125006A4 (en) | 2010-11-24 |
| EP2125006A2 (en) | 2009-12-02 |
| US7892824B2 (en) | 2011-02-22 |
| EP2125006B1 (en) | 2013-10-16 |
| US20080249052A1 (en) | 2008-10-09 |
| WO2008088895A3 (en) | 2008-10-30 |
| WO2008088895A2 (en) | 2008-07-24 |
| CN101711164B (zh) | 2014-06-04 |
| CN101711164A (zh) | 2010-05-19 |
| WO2008088895A9 (en) | 2008-09-18 |
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