BR112022009895A2 - Construtos de terapia de gene de microdistrofina e usos dos mesmos - Google Patents
Construtos de terapia de gene de microdistrofina e usos dos mesmosInfo
- Publication number
- BR112022009895A2 BR112022009895A2 BR112022009895A BR112022009895A BR112022009895A2 BR 112022009895 A2 BR112022009895 A2 BR 112022009895A2 BR 112022009895 A BR112022009895 A BR 112022009895A BR 112022009895 A BR112022009895 A BR 112022009895A BR 112022009895 A2 BR112022009895 A2 BR 112022009895A2
- Authority
- BR
- Brazil
- Prior art keywords
- gene therapy
- microdystrophin
- constructs
- therapy constructs
- microdystrophin gene
- Prior art date
Links
- 238000001415 gene therapy Methods 0.000 title abstract 3
- 108090000623 proteins and genes Proteins 0.000 abstract 3
- 210000004027 cell Anatomy 0.000 abstract 1
- 210000000663 muscle cell Anatomy 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 238000002560 therapeutic procedure Methods 0.000 abstract 1
- 239000013603 viral vector Substances 0.000 abstract 1
Classifications
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- 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
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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
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/62—DNA sequences coding for fusion proteins
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- 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
- A61K35/761—Adenovirus
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
- A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
- A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
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- 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
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- 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
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- 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
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- 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
-
- 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
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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
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2227/00—Animals characterised by species
- A01K2227/10—Mammal
- A01K2227/105—Murine
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2267/00—Animals characterised by purpose
- A01K2267/03—Animal model, e.g. for test or diseases
- A01K2267/035—Animal model for multifactorial diseases
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- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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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
- 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
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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
- 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
- C12N2750/14151—Methods of production or purification of viral material
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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
- 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
- C12N2750/14171—Demonstrated in vivo effect
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2800/00—Nucleic acids vectors
- C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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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
- 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
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- 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
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Organic Chemistry (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Pharmacology & Pharmacy (AREA)
- Biomedical Technology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Epidemiology (AREA)
- Gastroenterology & Hepatology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Virology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Toxicology (AREA)
- Physics & Mathematics (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Physical Education & Sports Medicine (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Immunology (AREA)
- Marine Sciences & Fisheries (AREA)
- Mycology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
Abstract
CONSTRUTOS DE TERAPIA DE GENE DE MICRODISTROFINA E USOS DOS MESMOS. É fornecida uma invenção baseada, em parte, em novos construtos de genes que codificam uma proteína de microdistrofina para uso em terapia de gene. Os construtos do gene da microdistrofina e os cassetes de expressão foram engenheirados para terapia melhorada em relação à eficácia, potência e segurança para o indivíduo quando expressos por um vetor viral em células musculares e/ou células do CNS.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201962941719P | 2019-11-28 | 2019-11-28 | |
US202063024933P | 2020-05-14 | 2020-05-14 | |
PCT/US2020/062484 WO2021108755A2 (en) | 2019-11-28 | 2020-11-27 | Microdystrophin gene therapy constructs and uses thereof |
Publications (1)
Publication Number | Publication Date |
---|---|
BR112022009895A2 true BR112022009895A2 (pt) | 2022-08-09 |
Family
ID=74206146
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
BR112022009895A BR112022009895A2 (pt) | 2019-11-28 | 2020-11-27 | Construtos de terapia de gene de microdistrofina e usos dos mesmos |
Country Status (11)
Country | Link |
---|---|
US (1) | US20230270886A1 (pt) |
EP (1) | EP4065596A2 (pt) |
JP (1) | JP2023503637A (pt) |
KR (1) | KR20220107222A (pt) |
AU (1) | AU2020392243A1 (pt) |
BR (1) | BR112022009895A2 (pt) |
CA (1) | CA3159516A1 (pt) |
CL (1) | CL2022001372A1 (pt) |
MX (1) | MX2022006427A (pt) |
TW (1) | TW202134260A (pt) |
WO (1) | WO2021108755A2 (pt) |
Families Citing this family (19)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AR121954A1 (es) | 2020-04-29 | 2022-07-27 | Bristol Myers Squibb Co | Distrofinas miniaturizadas con dominios de fusión de espectrina y usos de las mismas |
WO2022076750A2 (en) | 2020-10-07 | 2022-04-14 | Regenxbio Inc. | Recombinant adeno-associated viruses for cns or muscle delivery |
TW202309293A (zh) * | 2021-04-26 | 2023-03-01 | 美商銳進科斯生物股份有限公司 | 用於治療肌縮蛋白症(dystrophinopathy)之微小肌縮蛋白基因療法給藥 |
WO2022261143A2 (en) * | 2021-06-07 | 2022-12-15 | University Of Florida Research Foundation, Incorporated | Gene therapy for duchenne muscular dystrophy |
KR20240023643A (ko) * | 2021-06-23 | 2024-02-22 | 신프로믹스 엘티디 | 조절 핵산 서열 |
EP4373947A1 (en) * | 2021-07-19 | 2024-05-29 | New York University | Auf1 combination therapies for treatment of muscle degenerative disease |
WO2023018854A2 (en) * | 2021-08-11 | 2023-02-16 | Solid Biosciences Inc. | Treatment of muscular dystrophy |
WO2023019168A1 (en) * | 2021-08-11 | 2023-02-16 | Ultragenyx Pharmaceutical Inc. | Compositions and methods for treating a muscular dystrophy |
AU2022358523A1 (en) * | 2021-09-30 | 2024-03-14 | The Board Of Regents Of The Universityof Texas System | Slc13a5 gene therapy vectors and uses thereof |
WO2023060269A1 (en) | 2021-10-07 | 2023-04-13 | Regenxbio Inc. | Recombinant adeno-associated viruses for targeted delivery |
WO2023064947A1 (en) | 2021-10-15 | 2023-04-20 | Regenxbio Inc. | Antibodies and methods of using thereof |
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 |
WO2023141582A1 (en) | 2022-01-21 | 2023-07-27 | Regenxbio Inc. | Engineered promoters |
EP4230196A1 (en) | 2022-02-21 | 2023-08-23 | Som Innovation Biotech, S.A. | Compounds for use in the treatment of dystrophinopathies |
WO2023183623A1 (en) | 2022-03-25 | 2023-09-28 | Regenxbio Inc. | Dominant-negative tumor necrosis factor alpha adeno-associated virus gene therapy |
WO2023248251A1 (en) * | 2022-06-24 | 2023-12-28 | Indian Institute Of Technology Kanpur | An optimized aav vector for gene therapy of muscular dystrophy |
WO2024044725A2 (en) | 2022-08-24 | 2024-02-29 | Regenxbio Inc. | Recombinant adeno-associated viruses and uses thereof |
WO2024081746A2 (en) | 2022-10-11 | 2024-04-18 | Regenxbio Inc. | Engineered nucleic acid regulatory elements and methods and uses thereof |
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 |
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KR20230067694A (ko) | 2014-09-24 | 2023-05-16 | 시티 오브 호프 | 고효율 게놈 편집을 위한 아데노-관련 바이러스 벡터 변이체 및 이의 방법 |
AU2016206518B2 (en) * | 2015-01-16 | 2020-03-05 | University Of Washington | Novel micro-dystrophins and related methods 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 |
JP6665466B2 (ja) | 2015-09-26 | 2020-03-13 | 日亜化学工業株式会社 | 半導体発光素子及びその製造方法 |
WO2017070491A1 (en) | 2015-10-23 | 2017-04-27 | Applied Genetic Technologies Corporation | Ophthalmic formulations |
CA3056638A1 (en) * | 2017-03-17 | 2018-09-20 | Research Institute At Nationwide Children's Hospital | Adeno-associated virus vector delivery of muscle specific micro-dystrophin to treat muscular dystrophy |
US20210346519A1 (en) | 2018-02-07 | 2021-11-11 | Genethon | Hybrid regulatory elements |
-
2020
- 2020-11-27 TW TW109141932A patent/TW202134260A/zh unknown
- 2020-11-27 BR BR112022009895A patent/BR112022009895A2/pt unknown
- 2020-11-27 JP JP2022531343A patent/JP2023503637A/ja active Pending
- 2020-11-27 KR KR1020227021099A patent/KR20220107222A/ko active Search and Examination
- 2020-11-27 US US17/778,651 patent/US20230270886A1/en active Pending
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- 2020-11-27 MX MX2022006427A patent/MX2022006427A/es unknown
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2022
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EP4065596A2 (en) | 2022-10-05 |
US20230270886A1 (en) | 2023-08-31 |
KR20220107222A (ko) | 2022-08-02 |
MX2022006427A (es) | 2022-09-07 |
WO2021108755A2 (en) | 2021-06-03 |
AU2020392243A1 (en) | 2022-06-16 |
CL2022001372A1 (es) | 2023-01-13 |
TW202134260A (zh) | 2021-09-16 |
JP2023503637A (ja) | 2023-01-31 |
CA3159516A1 (en) | 2021-06-03 |
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