MX2017006652A - Vectores de virus adeno-asociado (aav) dirigidos al sistema nervioso central. - Google Patents
Vectores de virus adeno-asociado (aav) dirigidos al sistema nervioso central.Info
- Publication number
- MX2017006652A MX2017006652A MX2017006652A MX2017006652A MX2017006652A MX 2017006652 A MX2017006652 A MX 2017006652A MX 2017006652 A MX2017006652 A MX 2017006652A MX 2017006652 A MX2017006652 A MX 2017006652A MX 2017006652 A MX2017006652 A MX 2017006652A
- Authority
- MX
- Mexico
- Prior art keywords
- nervous system
- central nervous
- aav vectors
- vectors targeted
- targeted
- Prior art date
Links
- 239000013598 vector Substances 0.000 title abstract 5
- 210000003169 central nervous system Anatomy 0.000 title abstract 3
- 241000700605 Viruses Species 0.000 abstract 2
- 210000000234 capsid Anatomy 0.000 abstract 2
- 210000004248 oligodendroglia Anatomy 0.000 abstract 2
Classifications
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
-
- 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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- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/711—Natural deoxyribonucleic acids, i.e. containing only 2'-deoxyriboses attached to adenine, guanine, cytosine or thymine and having 3'-5' phosphodiester links
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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
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K9/00—Medicinal preparations characterised by special physical form
- A61K9/0012—Galenical forms characterised by the site of application
- A61K9/0014—Skin, i.e. galenical aspects of topical compositions
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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
-
- 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
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- 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
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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
- C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
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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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- 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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- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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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
- C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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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
- C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
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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
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14145—Special targeting system for viral vectors
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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
-
- 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)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Pharmacology & Pharmacy (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Biochemistry (AREA)
- General Engineering & Computer Science (AREA)
- Epidemiology (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Gastroenterology & Hepatology (AREA)
- Mycology (AREA)
- Immunology (AREA)
- Neurosurgery (AREA)
- Dermatology (AREA)
- Physical Education & Sports Medicine (AREA)
- Orthopedic Medicine & Surgery (AREA)
- Diabetes (AREA)
- Hematology (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462082897P | 2014-11-21 | 2014-11-21 | |
| US201562218857P | 2015-09-15 | 2015-09-15 | |
| PCT/US2015/061788 WO2016081811A1 (en) | 2014-11-21 | 2015-11-20 | Aav vectors targeted to the central nervous system |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2017006652A true MX2017006652A (es) | 2017-08-21 |
Family
ID=56014589
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2017006652A MX2017006652A (es) | 2014-11-21 | 2015-11-20 | Vectores de virus adeno-asociado (aav) dirigidos al sistema nervioso central. |
Country Status (12)
| Country | Link |
|---|---|
| US (4) | US10532110B2 (enExample) |
| EP (2) | EP3221453A4 (enExample) |
| JP (4) | JP2017536116A (enExample) |
| KR (2) | KR20240063169A (enExample) |
| CN (2) | CN114480440A (enExample) |
| AU (2) | AU2015349759B2 (enExample) |
| BR (1) | BR112017010447A2 (enExample) |
| CA (1) | CA2967393A1 (enExample) |
| IL (1) | IL252072B (enExample) |
| MX (1) | MX2017006652A (enExample) |
| RU (1) | RU2727015C2 (enExample) |
| WO (1) | WO2016081811A1 (enExample) |
Families Citing this family (69)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2015191508A1 (en) | 2014-06-09 | 2015-12-17 | Voyager Therapeutics, Inc. | Chimeric capsids |
| CN112553229A (zh) | 2014-11-05 | 2021-03-26 | 沃雅戈治疗公司 | 用于治疗帕金森病的aadc多核苷酸 |
| RU2020108189A (ru) | 2014-11-14 | 2020-03-11 | Вояджер Терапьютикс, Инк. | Композиции и способы лечения бокового амиотрофического склероза (als) |
| MX2017006217A (es) | 2014-11-14 | 2018-05-02 | Voyager Therapeutics Inc | Polinucleotidos moduladores. |
| BR112017010447A2 (pt) | 2014-11-21 | 2018-05-15 | The University Of North Carolina At Chapel Hill | vetores de aav direcionados ao sistema nervoso central |
| EP3230441A4 (en) | 2014-12-12 | 2018-10-03 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scaav |
| CN107249646B (zh) | 2014-12-16 | 2021-06-29 | 内布拉斯加大学董事会 | 用于青少年巴滕病的基因疗法 |
| GB201508026D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Capsid |
| US11701410B2 (en) | 2015-05-22 | 2023-07-18 | Cls Therapeutics Limited | Extracellular DNA as a therapeutic target in neurodegeneration |
| JP7064214B2 (ja) | 2015-09-28 | 2022-05-10 | 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 | 抗体回避性ウイルスベクターのための方法および組成物 |
| AU2016362477A1 (en) | 2015-12-02 | 2018-06-14 | Voyager Therapeutics, Inc. | Assays for the detection of AAV neutralizing antibodies |
| US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
| EP3448874A4 (en) | 2016-04-29 | 2020-04-22 | Voyager Therapeutics, Inc. | COMPOSITIONS FOR TREATING A DISEASE |
| AU2017267665C1 (en) | 2016-05-18 | 2023-10-05 | Voyager Therapeutics, Inc. | Modulatory polynucleotides |
| BR112018073472A2 (pt) | 2016-05-18 | 2019-08-27 | Voyager Therapeutics, Inc. | composições e métodos de tratamento da doença de huntington |
| WO2017218450A1 (en) | 2016-06-13 | 2017-12-21 | The University Of North Carolina At Chapel Hill | Optimized cln1 genes and expression cassettes and their use |
| MA44546B1 (fr) * | 2016-06-15 | 2021-03-31 | Univ California | Virus adéno-associés variants et procédés d'utilisation |
| JP7226801B2 (ja) * | 2016-08-16 | 2023-02-21 | 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 | 標的遺伝子導入のための方法および組成物 |
| WO2018044933A1 (en) | 2016-08-30 | 2018-03-08 | The Regents Of The University Of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
| CN111108198A (zh) | 2017-05-05 | 2020-05-05 | 沃雅戈治疗公司 | 治疗亨廷顿病的组合物和方法 |
| CA3061652A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
| JOP20190269A1 (ar) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | بولي نوكليوتيدات aadc لعلاج مرض باركنسون |
| CN111132626B (zh) | 2017-07-17 | 2024-01-30 | 沃雅戈治疗公司 | 轨迹阵列引导系统 |
| WO2019028306A2 (en) | 2017-08-03 | 2019-02-07 | Voyager Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR ADMINISTRATION OF ADENO-ASSOCIATED VIRUSES |
| AU2018338728B2 (en) | 2017-09-29 | 2025-01-02 | Centre National De La Recherche Scientifique (Cnrs) | Rescue of central and peripheral neurological phenotype of Friedreich's Ataxia by intravenous delivery |
| US20200237799A1 (en) | 2017-10-16 | 2020-07-30 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| CN111479924B (zh) | 2017-10-16 | 2024-06-14 | 沃雅戈治疗公司 | 肌萎缩性侧索硬化症(als)的治疗 |
| RU2020118342A (ru) | 2017-11-07 | 2021-12-08 | Дзе Юниверсити Оф Норт Каролина Эт Чепел Хилл | Оптимизированные гены aga и экспрессионные кластеры и их применение |
| EP3710010A4 (en) * | 2017-12-20 | 2021-09-22 | Ovid Therapeutics Inc. | USE OF HM4DI IN TREATMENT OF SEQUENCE DISEASES |
| AU2019209770B2 (en) | 2018-01-16 | 2025-07-31 | Cls Therapeutics Limited | Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity |
| JP7425738B2 (ja) | 2018-04-03 | 2024-01-31 | ギンコ バイオワークス インコーポレイテッド | 眼組織を標的とするウイルスベクター |
| MX2020010464A (es) * | 2018-04-03 | 2021-01-29 | Vectores de virus que evitan anticuerpos. | |
| US12091435B2 (en) | 2018-04-03 | 2024-09-17 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
| AU2019268330A1 (en) | 2018-05-15 | 2020-11-26 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of Parkinson's disease |
| CN108676097B (zh) * | 2018-05-24 | 2020-01-14 | 北京肽和生物科技有限公司 | 一种靶向肿瘤细胞的嵌合肽或嵌合蛋白及其应用 |
| AU2019287468B2 (en) * | 2018-06-12 | 2024-10-10 | The University Of North Carolina At Chapel Hill | Synthetic liver-tropic adeno-associated virus capsids and uses thereof |
| US12275948B2 (en) | 2018-06-28 | 2025-04-15 | The University Of North Carolina At Chapel Hill | Optimized CLN5 genes and expression cassettes and their use |
| IL280300B2 (en) | 2018-08-10 | 2025-06-01 | Univ North Carolina Chapel Hill | Optimized cln7 genes and expression cassettes and their use |
| CN113966399A (zh) | 2018-09-26 | 2022-01-21 | 加州理工学院 | 用于靶向基因疗法的腺相关病毒组合物 |
| EP3856762A1 (en) | 2018-09-28 | 2021-08-04 | Voyager Therapeutics, Inc. | Frataxin expression constructs having engineered promoters and methods of use thereof |
| CN119684417A (zh) | 2018-10-02 | 2025-03-25 | 沃雅戈治疗公司 | 重新定向aav衣壳的嗜性 |
| BR112021006074A2 (pt) | 2018-11-05 | 2021-07-20 | The University Of North Carolina At Chapel Hill | genes e cassetes de expressão fig4 otimizados e seu uso |
| MX2021006646A (es) * | 2018-12-05 | 2021-12-10 | Abeona Therapeutics Inc | Vector viral adenoasociado recombinante para el suministro de genes. |
| WO2020132385A1 (en) | 2018-12-21 | 2020-06-25 | The University Of North Carolina At Chapel Hill | Optimized galc genes and expression cassettes and their use |
| US12516351B2 (en) * | 2019-04-29 | 2026-01-06 | The Trustees Of The University Of Pennsylvania | AAV capsids and compositions containing same |
| CA3144654A1 (en) | 2019-04-29 | 2020-11-05 | The University Of North Carolina At Chapel Hill | Optimized sumf1 genes and expression cassettes and their use |
| AU2020279387A1 (en) | 2019-05-22 | 2021-12-09 | The University Of North Carolina At Chapel Hill | UBE3A genes and expression cassettes and their use |
| WO2021011368A1 (en) * | 2019-07-12 | 2021-01-21 | Dmitry Dmitrievich Genkin | Treatment of diseases associated with protein misfolding by nervous system expression of an enzyme which has a deoxyribonuclease (dnase) activity |
| JP2022545534A (ja) * | 2019-08-29 | 2022-10-27 | ザ ボード オブ トラスティーズ オブ ザ レランド スタンフォード ジュニア ユニバーシティー | ヒト血液脳関門を通過するためのアデノ随伴ウイルスベクター |
| US20220324921A1 (en) * | 2019-09-03 | 2022-10-13 | University Of Cincinnati | Methods and compositions for the treatment of als |
| IL292297A (en) | 2019-10-17 | 2022-06-01 | Stridebio Inc | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
| CN115210248A (zh) * | 2020-02-18 | 2022-10-18 | 北卡罗来纳大学查佩尔希尔分校 | Aav衣壳-启动子相互作用和细胞选择性基因表达 |
| CA3176506A1 (en) * | 2020-05-01 | 2021-11-04 | The Broad Institute, Inc. | Engineered central nervous system compositions |
| WO2021225921A1 (en) * | 2020-05-04 | 2021-11-11 | The University Of North Carolina At Chapel Hill | Modified adeno-associated virus vectors that evade neutralizing antibodies and uses thereof |
| WO2021226008A1 (en) * | 2020-05-05 | 2021-11-11 | The University Of North Carolina At Chapel Hill | Modified adeno-associated virus 5 capsids and uses thereof |
| IL298001A (en) | 2020-05-13 | 2023-01-01 | Voyager Therapeutics Inc | Redirecting tropism of adeno-associated virus capsids |
| CN115806596B (zh) * | 2020-05-22 | 2026-03-06 | 中国医学科学院血液病医院(中国医学科学院血液学研究所) | 腺相关病毒突变体及其应用 |
| WO2022011390A1 (en) | 2020-07-08 | 2022-01-13 | Baylor College Of Medicine | Gene therapy for stxbp1 encephalopathy |
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| US11491242B2 (en) | 2022-11-08 |
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| EP3221453A1 (en) | 2017-09-27 |
| RU2727015C2 (ru) | 2020-07-17 |
| CA2967393A1 (en) | 2016-05-26 |
| AU2015349759A1 (en) | 2017-05-25 |
| KR20240063169A (ko) | 2024-05-10 |
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| EP3221453A4 (en) | 2018-09-12 |
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| WO2016081811A1 (en) | 2016-05-26 |
| RU2017121593A (ru) | 2018-12-21 |
| US10532110B2 (en) | 2020-01-14 |
| NZ731673A (en) | 2024-07-26 |
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