MA47800A - Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire - Google Patents
Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaireInfo
- Publication number
- MA47800A MA47800A MA047800A MA47800A MA47800A MA 47800 A MA47800 A MA 47800A MA 047800 A MA047800 A MA 047800A MA 47800 A MA47800 A MA 47800A MA 47800 A MA47800 A MA 47800A
- Authority
- MA
- Morocco
- Prior art keywords
- dystrophine
- adeno
- fragment
- micro
- viral vector
- 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
-
- 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
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Medicinal Chemistry (AREA)
- Organic Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Epidemiology (AREA)
- Zoology (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biophysics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- Virology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (1)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201762473255P | 2017-03-17 | 2017-03-17 |
Publications (1)
Publication Number | Publication Date |
---|---|
MA47800A true MA47800A (fr) | 2020-01-22 |
Family
ID=64564915
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MA047800A MA47800A (fr) | 2017-03-17 | 2018-03-16 | Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire |
Country Status (5)
Country | Link |
---|---|
US (2) | US11338045B2 (fr) |
EP (1) | EP3596112A2 (fr) |
JP (2) | JP7162021B2 (fr) |
MA (1) | MA47800A (fr) |
WO (1) | WO2019012336A2 (fr) |
Families Citing this family (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
MA47800A (fr) * | 2017-03-17 | 2020-01-22 | Univ Newcastle | Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire |
KR20220119703A (ko) * | 2019-12-24 | 2022-08-30 | 아스클레피오스 바이오파마슈티컬, 인크. | 조절 핵산 서열 |
WO2021222332A1 (fr) | 2020-04-29 | 2021-11-04 | Bristol-Myers Squibb Company | Dystrophines miniaturisées ayant des domaines de fusion de spectrine et leurs utilisations |
EP4108263A3 (fr) * | 2021-06-02 | 2023-03-22 | Research Institute at Nationwide Children's Hospital | Produits de virus adéno-associés recombinants et méthodes de traitement de la dystrophie musculaire des ceintures 2a |
TW202337476A (zh) * | 2022-01-17 | 2023-10-01 | 瑞士商迪納柯公司 | 用於與肌肉萎縮蛋白有關之心肌病之基因療法組合物及治療 |
WO2023212193A1 (fr) * | 2022-04-27 | 2023-11-02 | The Curators Of The University Of Missouri | Micro-dystrophine pour protection cardiaque |
CN115838725B (zh) * | 2022-12-30 | 2023-09-08 | 广州派真生物技术有限公司 | 在哺乳动物心脏中特异性启动基因的启动子序列及其应用 |
Family Cites Families (30)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
WO1995013365A1 (fr) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Production de titres eleves de vecteurs d'aav recombinants |
JP3952312B2 (ja) | 1993-11-09 | 2007-08-01 | メディカル カレッジ オブ オハイオ | アデノ関連ウイルス複製遺伝子を発現可能な安定な細胞株 |
US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
WO1996017947A1 (fr) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Lignees cellulaires d'encapsidation utilisees pour la generation de titres hauts de vecteurs aav recombinants |
FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
CA2625279A1 (fr) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Purification d'adenovirus et de virus adeno-associe (aav) par voie chromatographique |
AU715543B2 (en) | 1995-09-08 | 2000-02-03 | Genzyme Corporation | Improved AAV vectors for gene therapy |
US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
EP0932418B1 (fr) | 1996-09-06 | 2007-12-05 | The Trustees Of The University Of Pennsylvania | Methode de therapie genique basee sur des virus adeno-associes de recombinaison |
US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
CA2830694C (fr) | 1997-09-05 | 2018-02-27 | Genzyme Corporation | Procedes de generation de preparations de vecteurs de aav recombinants dont le titre est eleve et qui sont exemptes de virus assistant |
JPH11318467A (ja) * | 1998-05-08 | 1999-11-24 | Japan Science & Technology Corp | 短縮型ジストロフィン |
US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
CA2406743A1 (fr) | 2000-04-28 | 2001-11-08 | The Trustees Of The University Of Pennsylvania | Vecteurs aav recombinants dotes de capsides aav5 et vecteurs aav5 pseudotypes dans des capsides heterologues |
US6962815B2 (en) | 2001-01-05 | 2005-11-08 | Children's Hopital Inc. | AAV2 vectors and methods |
PT1453547T (pt) | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
US20080044393A1 (en) | 2004-07-16 | 2008-02-21 | White Robert L | Retinal dystrophin transgene and methods of use thereof |
WO2013016352A1 (fr) | 2011-07-25 | 2013-01-31 | Nationwide Children's Hospital, Inc. | Produits viraux recombinants et procédés pour inhibition de l'expression de dux4 |
DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
JP6202701B2 (ja) | 2014-03-21 | 2017-09-27 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及びプログラム |
EP2960336A1 (fr) | 2014-06-27 | 2015-12-30 | Genethon | Traitement systémique efficace de pathologies dystrophiques musculaires |
JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
IL286316B2 (en) * | 2015-01-16 | 2023-03-01 | Univ Washington | New micro-dystrophins and related method of use |
GB201507842D0 (en) | 2015-05-07 | 2015-06-17 | New Royal Holloway & Bedford | Production of large-sized microdystrophins in an AAV-based vector configuration |
WO2017165859A1 (fr) | 2016-03-24 | 2017-09-28 | Research Institute At Nationwide Children's Hospital | Protéines de capside virale modifiées |
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 |
MA47800A (fr) * | 2017-03-17 | 2020-01-22 | Univ Newcastle | Délivrance par vecteur viral adéno-associé d'un fragment de micro-dystrophine pour traiter la dystrophie musculaire |
-
2018
- 2018-03-16 MA MA047800A patent/MA47800A/fr unknown
- 2018-03-16 EP EP18811911.9A patent/EP3596112A2/fr active Pending
- 2018-03-16 JP JP2019571814A patent/JP7162021B2/ja active Active
- 2018-03-16 WO PCT/IB2018/001201 patent/WO2019012336A2/fr active Application Filing
- 2018-03-16 US US16/494,645 patent/US11338045B2/en active Active
-
2022
- 2022-05-23 US US17/751,195 patent/US20230049491A1/en active Pending
- 2022-10-17 JP JP2022166267A patent/JP7493566B2/ja active Active
Also Published As
Publication number | Publication date |
---|---|
WO2019012336A3 (fr) | 2019-03-07 |
US20230049491A1 (en) | 2023-02-16 |
JP2022185154A (ja) | 2022-12-13 |
US11338045B2 (en) | 2022-05-24 |
WO2019012336A2 (fr) | 2019-01-17 |
JP7162021B2 (ja) | 2022-10-27 |
EP3596112A2 (fr) | 2020-01-22 |
JP7493566B2 (ja) | 2024-05-31 |
US20200078473A1 (en) | 2020-03-12 |
JP2020510447A (ja) | 2020-04-09 |
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