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)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Biophysics (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- Cell Biology (AREA)
- Epidemiology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Gastroenterology & Hepatology (AREA)
- Toxicology (AREA)
- Cardiology (AREA)
- Mycology (AREA)
- Hospice & Palliative Care (AREA)
- Heart & Thoracic Surgery (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 (fr) | 2007-01-18 | 2008-01-18 | Mini/microgènes synthétiques de la dystrophine pour redonner de la no-synthase neuronale au sarcolemme |
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 (fr) |
| EP (1) | EP2125006B1 (fr) |
| JP (1) | JP5575486B2 (fr) |
| CN (1) | CN101711164B (fr) |
| HK (1) | HK1143326A1 (fr) |
| WO (1) | WO2008088895A2 (fr) |
Families Citing this family (61)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2006213686A1 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method 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 |
| EP2396343B1 (fr) | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Vecteurs viraux modifiés et procédés de fabrication et d'utilisation de ceux-ci |
| EP2524037B1 (fr) | 2010-01-12 | 2018-05-16 | The University Of North Carolina At Chapel Hill | Répétitions terminales inversées restrictives pour vecteurs viraux |
| US9169492B2 (en) | 2010-02-05 | 2015-10-27 | The University Of North Carolina At Chapel Hill | Compositions and methods for enhanced parvovirus transduction |
| WO2012109570A1 (fr) | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Vecteurs viraux à profil de transduction modifiée et procédés pour les préparer et les utiliser |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| WO2013075008A1 (fr) * | 2011-11-16 | 2013-05-23 | University Of Florida Research Foundation Inc. | Systèmes de vecteur double aav pour une thérapie génique |
| CN103405774A (zh) * | 2012-10-24 | 2013-11-27 | 尹海芳 | 一种新型药物靶向运输辅助剂 |
| US20140140969A1 (en) * | 2012-11-20 | 2014-05-22 | Sangamo Biosciences, Inc. | 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 |
| AU2014227766B2 (en) | 2013-03-15 | 2018-10-04 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding AAV vectors |
| EP3517612A1 (fr) | 2013-03-15 | 2019-07-31 | The University of North Carolina At Chapel Hill | Répétitions terminales inversées de synthèse du virus adéno-associé |
| CA2972807C (fr) | 2015-01-14 | 2024-01-16 | The University Of North Carolina At Chapel Hill | Procedes et compositions destines au transfert de genes cible |
| NZ734019A (en) | 2015-01-16 | 2023-01-27 | Univ Washington | Novel micro-dystrophins and related methods of use |
| WO2016134300A1 (fr) | 2015-02-20 | 2016-08-25 | Board Of Regents, The University Of Texas System | Méthodes et compositions permettant d'utiliser la chlamydia atténuée en tant que vaccin et vecteur |
| GB201507842D0 (en) * | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
| CA2996420A1 (fr) | 2015-09-28 | 2017-04-06 | The University Of North Carolina At Chapel Hill | Procedes et compositions pour vecteurs viraux evitant les anticorps |
| WO2017062835A2 (fr) | 2015-10-09 | 2017-04-13 | Sarepta Therapeutics, Inc. | Compositions et méthodes pour traiter la dystrophie musculaire de duchenne et troubles associés |
| 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 |
| CN109121395B (zh) | 2016-04-15 | 2022-08-09 | 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 |
| EP4011206B1 (fr) | 2016-06-21 | 2023-11-01 | The Curators of the University of Missouri | Protéines de la dystrophine modifiées |
| CA2971303A1 (fr) * | 2016-06-21 | 2017-12-21 | Bamboo Therapeutics, Inc. | Genes de mini-dystrophine optimises et cassettes d'expression et leur utilisation |
| EP3491008A2 (fr) | 2016-07-26 | 2019-06-05 | BioMarin Pharmaceutical Inc. | Nouvelles protéines de capside de virus adéno-associés |
| WO2018035451A1 (fr) | 2016-08-19 | 2018-02-22 | Calimmune, Inc. | Méthodes et compositions pour le traitement d'affections à l'aide d'un virus adéno-associé recombinant auto-complémentaire |
| WO2018106956A2 (fr) | 2016-12-07 | 2018-06-14 | University Of Florida Research Foundation, Incorporated | Adnc d'il-1ra |
| US11541131B2 (en) | 2017-03-10 | 2023-01-03 | Genethon | Treatment of glycogen storage disease III |
| JP7132934B2 (ja) | 2017-03-15 | 2022-09-07 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | ポリプロイド性アデノ随伴ウイルスベクターならびにその作製および使用の方法 |
| US11534501B2 (en) | 2017-10-18 | 2022-12-27 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
| JP7406677B2 (ja) | 2018-04-03 | 2023-12-28 | ギンコ バイオワークス インコーポレイテッド | 抗体を回避するウイルスベクター |
| CA3095179A1 (fr) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Vecteurs viraux evitant les anticorps |
| KR20210006358A (ko) | 2018-04-03 | 2021-01-18 | 스트라이드바이오 인코포레이티드 | 안 조직을 표적으로 하기 위한 바이러스 벡터 |
| US11660353B2 (en) | 2018-04-27 | 2023-05-30 | Decibel Therapeutics, Inc. | Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems |
| EP3790627A2 (fr) | 2018-05-09 | 2021-03-17 | BioMarin Pharmaceutical Inc. | Méthodes de traitement de la grippe |
| 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 |
| EP3807413A1 (fr) * | 2018-06-18 | 2021-04-21 | Research Institute at Nationwide Children's Hospital | Administration par vecteur de virus adéno-associé de micro-dystrophine spécifique des muscles pour traiter la dystrophie musculaire |
| CN112639110A (zh) | 2018-06-22 | 2021-04-09 | 阿斯克肋匹奥生物制药公司 | 用于基因递送以在细胞内持续存在的载体 |
| 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 |
| JP2022534119A (ja) | 2019-05-30 | 2022-07-27 | ソリッド・バイオサイエンシーズ・インコーポレーテッド | 組換えヘルペスウイルスベクター |
| JP2022540819A (ja) * | 2019-07-10 | 2022-09-20 | ユニバーシティ オブ ワシントン | 非ウイルス性免疫標的化 |
| TW202128736A (zh) | 2019-10-17 | 2021-08-01 | 美商史崔德生物公司 | 用於治療ｃ型尼曼—匹克病之腺相關病毒載體 |
| 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 |
| BR112022009895A2 (pt) * | 2019-11-28 | 2022-08-09 | Regenxbio Inc | Construtos de terapia de gene de microdistrofina e usos dos mesmos |
| IL297753A (en) * | 2020-04-29 | 2022-12-01 | Bristol Myers Squibb Co | Minimized dystrophins with spectrin fusion domains and uses thereof |
| WO2022076750A2 (fr) | 2020-10-07 | 2022-04-14 | Regenxbio Inc. | Virus adéno-associés recombinants pour administration musculaire ou dans le snc |
| AU2021369480A1 (en) | 2020-10-28 | 2023-04-20 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding AAV2.5 vector |
| WO2022093769A1 (fr) | 2020-10-28 | 2022-05-05 | The University Of North Carolina At Chapel Hill | Procédés et compositions de double liaison glycane de vecteurs de vaa2.5 |
| 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 |
| WO2022216749A1 (fr) | 2021-04-05 | 2022-10-13 | Solid Biosciences Inc. | Vecteur du virus de l'herpès simplex recombinant |
| US20240189447A1 (en) | 2021-04-16 | 2024-06-13 | Asklepios Biopharmaceutical, Inc. | Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response |
| JP2024517143A (ja) * | 2021-04-26 | 2024-04-19 | リジェネックスバイオ インコーポレイテッド | ジストロフィン異常症の治療のためのマイクロジストロフィン遺伝子療法投与 |
| WO2023056311A1 (fr) * | 2021-09-28 | 2023-04-06 | University Of Florida Research Foundation, Incorporated | Thérapie génique pour dystrophie musculaire de duchenne |
| WO2023060269A1 (fr) | 2021-10-07 | 2023-04-13 | Regenxbio Inc. | Virus adéno-associés recombinants pour administration ciblée |
| WO2023135316A1 (fr) * | 2022-01-17 | 2023-07-20 | Dinaqor Ag | Composition de thérapie génique et traitement de cardiomyopathie liée à la dystrophine |
| EP4215614A1 (fr) | 2022-01-24 | 2023-07-26 | Dynacure | Thérapie de combinaison pour maladies liées à la dystrophine |
| WO2023150687A1 (fr) | 2022-02-04 | 2023-08-10 | Ginkgo Bioworks, Inc. | Vecteurs viraux adéno-associés recombinants et méthodes d'utilisation associées |
| WO2023212193A1 (fr) * | 2022-04-27 | 2023-11-02 | The Curators Of The University Of Missouri | Micro-dystrophine pour protection cardiaque |
| WO2024124019A2 (fr) | 2022-12-07 | 2024-06-13 | Ginkgo Bioworks, Inc. | Vecteurs aav ciblant des cellules souches hématopoïétiques |
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 |
| WO2002029056A2 (fr) * | 2000-10-06 | 2002-04-11 | Regents Of The University Of Michigan | Sequences peptidiques et d'acide nucleique de mini-dystrophine |
-
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 WO PCT/US2008/000717 patent/WO2008088895A2/fr active Application Filing
- 2008-01-18 EP EP08724643.5A patent/EP2125006B1/fr active Active
- 2008-01-18 US US12/009,537 patent/US7892824B2/en active Active
-
2010
- 2010-10-20 HK HK10109944.6A patent/HK1143326A1/xx unknown
Also Published As
| Publication number | Publication date |
|---|---|
| CN101711164A (zh) | 2010-05-19 |
| US7892824B2 (en) | 2011-02-22 |
| JP2010516252A (ja) | 2010-05-20 |
| JP5575486B2 (ja) | 2014-08-20 |
| US20080249052A1 (en) | 2008-10-09 |
| EP2125006A2 (fr) | 2009-12-02 |
| WO2008088895A3 (fr) | 2008-10-30 |
| WO2008088895A2 (fr) | 2008-07-24 |
| EP2125006B1 (fr) | 2013-10-16 |
| EP2125006A4 (fr) | 2010-11-24 |
| WO2008088895A9 (fr) | 2008-09-18 |
| CN101711164B (zh) | 2014-06-04 |
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