IN2012DN06629A - - Google Patents
Info
- Publication number
- IN2012DN06629A IN2012DN06629A IN6629DEN2012A IN2012DN06629A IN 2012DN06629 A IN2012DN06629 A IN 2012DN06629A IN 6629DEN2012 A IN6629DEN2012 A IN 6629DEN2012A IN 2012DN06629 A IN2012DN06629 A IN 2012DN06629A
- Authority
- IN
- India
- Prior art keywords
- formulations
- aav
- superior
- methods
- clinical use
- Prior art date
Links
Classifications
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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
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K39/00—Medicinal preparations containing antigens or antibodies
- A61K39/12—Viral antigens
- A61K39/23—Parvoviridae, e.g. feline panleukopenia virus
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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
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
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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
- A61K48/0091—Purification or manufacturing processes for gene therapy compositions
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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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
- A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
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- C—CHEMISTRY; METALLURGY
- C07—ORGANIC CHEMISTRY
- C07K—PEPTIDES
- C07K2/00—Peptides of undefined number of amino acids; Derivatives 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
- 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
- C12N15/64—General methods for preparing the vector, for introducing it into the cell or for selecting the vector-containing 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
- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
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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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- 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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- 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
- C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
- C12N15/8645—Adeno-associated virus
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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
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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
- C12N2750/14051—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
- 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
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y301/00—Hydrolases acting on ester bonds (3.1)
- C12Y301/01—Carboxylic ester hydrolases (3.1.1)
- C12Y301/01064—Retinoid isomerohydrolase (3.1.1.64)
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Medicinal Chemistry (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Epidemiology (AREA)
- Immunology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Manufacturing & Machinery (AREA)
- Cell Biology (AREA)
- Gastroenterology & Hepatology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- General Chemical & Material Sciences (AREA)
- Mycology (AREA)
- Diabetes (AREA)
- Hematology (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US29918410P | 2010-01-28 | 2010-01-28 | |
PCT/US2011/022371 WO2011094198A1 (fr) | 2010-01-28 | 2011-01-25 | Plateforme de fabrication évolutive pour purification de vecteurs viraux et vecteurs viraux ainsi purifiés utilisables en thérapie génique |
Publications (1)
Publication Number | Publication Date |
---|---|
IN2012DN06629A true IN2012DN06629A (fr) | 2015-10-23 |
Family
ID=44319714
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
IN6629DEN2012 IN2012DN06629A (fr) | 2010-01-28 | 2011-01-25 |
Country Status (14)
Country | Link |
---|---|
US (4) | US9408904B2 (fr) |
EP (1) | EP2529020B1 (fr) |
JP (4) | JP2013517798A (fr) |
CN (2) | CN102947453A (fr) |
AU (1) | AU2011209743B2 (fr) |
BR (1) | BR112012018899A2 (fr) |
CA (1) | CA2787827C (fr) |
DK (1) | DK2529020T3 (fr) |
ES (1) | ES2680915T3 (fr) |
IL (1) | IL221158B (fr) |
IN (1) | IN2012DN06629A (fr) |
MX (1) | MX340102B (fr) |
PT (1) | PT2529020T (fr) |
WO (1) | WO2011094198A1 (fr) |
Families Citing this family (98)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
WO2010138263A2 (fr) | 2009-05-28 | 2010-12-02 | University Of Massachusetts | Nouveaux virus adéno-associés (aav) et leurs utilisations |
IN2012DN06629A (fr) * | 2010-01-28 | 2015-10-23 | Philadelphia Children Hospital | |
JP5963743B2 (ja) | 2010-04-23 | 2016-08-03 | ユニバーシティ オブ マサチューセッツ | Cnsターゲティングaavベクターおよびその使用方法 |
CA3050894C (fr) | 2010-04-23 | 2022-10-18 | University Of Massachusetts | Constructions d'expression a cistrons multiples |
WO2013036118A1 (fr) * | 2011-09-08 | 2013-03-14 | Uniqure Ip B.V. | Élimination de virus contaminants à partir de préparations de virus adéno-associés (vaa) |
FR3002237B1 (fr) * | 2013-02-15 | 2017-12-15 | Genethon | Methodes pour la production de particules virales aav double brin |
JP6649265B2 (ja) | 2013-11-26 | 2020-02-19 | ザ ユナイテッド ステイツ オブ アメリカ, アズ リプレゼンテッド バイ ザ セクレタリー, デパートメント オブ ヘルス アンド ヒューマン サービシーズ | 糖原病の処置のためのアデノ随伴ウイルスベクター |
GB201401707D0 (en) | 2014-01-31 | 2014-03-19 | Sec Dep For Health The | Adeno-associated viral vectors |
WO2015127128A2 (fr) | 2014-02-19 | 2015-08-27 | University Of Massachusetts | Vaa recombinants ayant des propriétés de transcytose utiles |
US10280418B2 (en) | 2014-03-18 | 2019-05-07 | Univeristy Of Massachusetts | RAAV-based compositions and methods for treating amyotrophic lateral sclerosis |
CR20160480A (es) * | 2014-03-21 | 2016-12-14 | Genzyme Corp | Terapia genetica para la retinitis pigmentaria |
EP3134431B1 (fr) | 2014-04-25 | 2021-04-07 | The Trustees Of The University Of Pennsylvania | Variants de ldlr et leur utilisation dans des compositions permettant de réduire les taux de cholestérol |
EP3134522B1 (fr) * | 2014-04-25 | 2021-10-06 | University of Massachusetts | Vecteurs de virus adéno-associés recombinants utiles pour réduire une immunité contre des produits transgéniques |
WO2015187825A2 (fr) | 2014-06-03 | 2015-12-10 | University Of Massachusetts | Compositions et méthodes de modulation de l'expression de la dysferline |
JP6842410B2 (ja) | 2014-10-03 | 2021-03-17 | ユニバーシティ オブ マサチューセッツ | 新規の高効率ライブラリーにより同定されるaavベクター |
US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
EP3209311B1 (fr) | 2014-10-21 | 2024-03-06 | University of Massachusetts | Variants de vaa recombinants et leurs utilisations |
CN107208142B (zh) * | 2014-11-28 | 2024-03-26 | 优尼科Ip有限公司 | 包含细小病毒病毒体的组合物中的dna杂质 |
EP3236984B1 (fr) | 2014-12-23 | 2020-07-22 | The U.S.A. as represented by the Secretary, Department of Health and Human Services | Vecteurs à base de virus adéno-associé codant pour une g6pc modifiée, et utilisations de ces derniers |
CN107428799A (zh) | 2015-01-13 | 2017-12-01 | 阿尔法韦士曼公司 | 纯化腺相关病毒(AAV)及/或重组腺相关病毒(rAAV)之方法及其梯度和流通式缓冲液 |
EP3054007A1 (fr) * | 2015-02-09 | 2016-08-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Purification de particules de virus adéno-associés de recombinaison comprenant une étape de purification par affinité immunologique |
EP3054006A1 (fr) | 2015-02-09 | 2016-08-10 | Institut National De La Sante Et De La Recherche Medicale (Inserm) | Purification de particule de virus adéno-associé recombinant avec une chromatographie à échange d'anions en plusieurs étapes |
EP3256170B1 (fr) | 2015-02-13 | 2020-09-23 | University of Massachusetts | Compositions et procédés pour l'administration transitoire de nucléases |
US20180245073A1 (en) * | 2015-02-23 | 2018-08-30 | Voyager Therapeutics, Inc. | Regulatable expression using adeno-associated virus (aav) |
CN115074366A (zh) | 2015-04-16 | 2022-09-20 | 埃默里大学 | 用于肝脏中蛋白质表达的重组启动子和载体及其用途 |
KR20180002706A (ko) | 2015-04-23 | 2018-01-08 | 워싱턴 스테이트 유니버시티 | Smad7 유전자 전달 치료법 |
CA3021949C (fr) | 2015-04-24 | 2023-10-17 | University Of Massachusetts | Constructions d'aav modifiees et utilisations de celles-ci |
WO2017070525A1 (fr) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Procédés et compositions pour le traitement du déséquilibre métabolique dans une maladie neurodégénérative |
EP3364996B1 (fr) | 2015-10-22 | 2021-08-25 | University of Massachusetts | Vecteurs du sérotype de virus adéno-associés ciblant la prostate |
CN105420275A (zh) * | 2015-11-27 | 2016-03-23 | 中国科学院苏州生物医学工程技术研究所 | 制备外源功能基因定点整合的人神经干细胞的方法 |
EP3400304B1 (fr) | 2015-12-11 | 2022-04-06 | The Trustees Of The University Of Pennsylvania | Thérapie génique pour le traitement de l'hypercholestérolémie familiale |
US11028372B2 (en) * | 2015-12-11 | 2021-06-08 | The Trustees Of The University Of Pennsylvania | Scalable purification method for AAVRH10 |
WO2017160360A2 (fr) | 2015-12-11 | 2017-09-21 | The Trustees Of The University Of Pennsylvania | Méthode de purification évolutive pour virus adéno-associé 9 (aav9) |
ES2934848T3 (es) * | 2015-12-11 | 2023-02-27 | Univ Pennsylvania | Método de purificación escalable para AAV8 |
WO2017100674A1 (fr) * | 2015-12-11 | 2017-06-15 | The Trustees Of The University Of Pennsylvania | Procédé de purification évolutif pour virus adéno-associé (aav) 1 |
JP7128741B2 (ja) | 2015-12-18 | 2022-08-31 | サンガモ セラピューティクス, インコーポレイテッド | T細胞受容体の標的化破壊 |
EA201891212A1 (ru) | 2015-12-18 | 2019-01-31 | Сангамо Терапьютикс, Инк. | Адресная дезорганизация клеточного рецептора гкгс |
US11826433B2 (en) | 2016-02-02 | 2023-11-28 | University Of Massachusetts | Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system |
WO2017136049A1 (fr) | 2016-02-02 | 2017-08-10 | Sangamo Biosciences, Inc. | Compositions pour lier des domaines de liaison à l'adn et des domaines de clivage |
WO2017136202A1 (fr) | 2016-02-05 | 2017-08-10 | Emory University | Injection de virus adéno-associé simple brin ou auto-complémentaire 9 dans le liquide cérébrospinal |
WO2017139643A1 (fr) | 2016-02-12 | 2017-08-17 | University Of Massachusetts | Agents thérapeutiques à base de micro-arn anti-angiogéniques pour l'inhibition de la néovascularisation cornéenne |
AU2017240721B2 (en) * | 2016-03-31 | 2023-10-12 | Spark Therapeutics, Inc. | Column-based fully scalable rAAV manufacturing process |
EP3440210A4 (fr) | 2016-04-05 | 2019-11-27 | University of Massachusetts | Compositions et procédés pour l'inhibition sélective de l'expression d'une protéine de type grainhead |
US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
US11882815B2 (en) | 2016-06-15 | 2024-01-30 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
GB201612248D0 (en) | 2016-07-14 | 2016-08-31 | Puridify Ltd | New process |
IL301963A (en) * | 2016-07-21 | 2023-06-01 | Spark Therapeutics Inc | Scalable High Recovery Methods for High-Throughput Recombinant Adeno-Associated Viral Vector Production and Recombinant Adeno-Associated Viral Vectors Made So |
WO2018022511A1 (fr) | 2016-07-25 | 2018-02-01 | The Trustees Of The University Of Pennsylvania | Compositions comprenant un variant de la lécithine-cholestérol-acyl-transférase et leurs utilisations |
EP3510161A4 (fr) | 2016-08-23 | 2020-04-22 | Akouos, Inc. | Compositions et méthodes pour traiter une déficience auditive non associée à l'âge chez un sujet humain |
SG10202007392SA (en) | 2016-08-24 | 2020-09-29 | Sangamo Therapeutics Inc | Engineered target specific nucleases |
IL264639B2 (en) | 2016-08-24 | 2024-01-01 | Sangamo Therapeutics Inc | Regulation of globulin gene expression using transgenic nucleases with zinc neurites |
US10457940B2 (en) | 2016-09-22 | 2019-10-29 | University Of Massachusetts | AAV treatment of Huntington's disease |
KR20190075964A (ko) | 2016-10-13 | 2019-07-01 | 유니버시티 오브 매사추세츠 | Aav 캡시드 설계 |
IL266162B2 (en) * | 2016-11-04 | 2023-11-01 | Baxalta Inc | Adeno-associated virus purification methods |
EP3321357A1 (fr) * | 2016-11-09 | 2018-05-16 | Deutsches Krebsforschungszentrum | Procédé évolutif de production et de purification de parvovirus oncolytique h-1 du rat à base d'élimination de particules vides par points isoélectriques |
US20190367944A1 (en) | 2017-01-30 | 2019-12-05 | The U.S.A., As Represented By The Secretary, Department Of Health And Human Services | Recombinant virus vectors for the treatment of glycogen storage disease |
US11649288B2 (en) | 2017-02-07 | 2023-05-16 | Seattle Children's Hospital | Phospholipid ether (PLE) CAR T cell tumor targeting (CTCT) agents |
EP3595688A4 (fr) | 2017-02-20 | 2020-12-30 | The Trustees Of The University Of Pennsylvania | Thérapie génique pour traiter l'hypercholestérolémie familiale |
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- 2011-01-25 AU AU2011209743A patent/AU2011209743B2/en active Active
- 2011-01-25 WO PCT/US2011/022371 patent/WO2011094198A1/fr active Application Filing
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- 2011-01-25 PT PT117375089T patent/PT2529020T/pt unknown
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AU2011209743A1 (en) | 2012-08-23 |
WO2011094198A1 (fr) | 2011-08-04 |
BR112012018899A2 (pt) | 2015-09-15 |
JP2016185153A (ja) | 2016-10-27 |
US20130072548A1 (en) | 2013-03-21 |
US20200405845A1 (en) | 2020-12-31 |
CN105838737A (zh) | 2016-08-10 |
DK2529020T3 (en) | 2018-08-06 |
EP2529020B1 (fr) | 2018-04-25 |
CA2787827C (fr) | 2020-11-10 |
CN102947453A (zh) | 2013-02-27 |
EP2529020A4 (fr) | 2013-10-16 |
ES2680915T3 (es) | 2018-09-11 |
JP2013517798A (ja) | 2013-05-20 |
AU2011209743B2 (en) | 2016-03-10 |
IL221158B (en) | 2018-07-31 |
US9408904B2 (en) | 2016-08-09 |
US20160206706A1 (en) | 2016-07-21 |
US10328145B2 (en) | 2019-06-25 |
EP2529020A1 (fr) | 2012-12-05 |
US20190321463A1 (en) | 2019-10-24 |
JP2018121653A (ja) | 2018-08-09 |
MX340102B (es) | 2016-06-24 |
US11878056B2 (en) | 2024-01-23 |
PT2529020T (pt) | 2018-07-30 |
CA2787827A1 (fr) | 2011-08-04 |
JP2018046820A (ja) | 2018-03-29 |
MX2012008757A (es) | 2012-11-06 |
JP6991095B2 (ja) | 2022-01-12 |
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