CA2942776C - Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter - Google Patents

Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter Download PDF

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CA2942776C
CA2942776C CA2942776A CA2942776A CA2942776C CA 2942776 C CA2942776 C CA 2942776C CA 2942776 A CA2942776 A CA 2942776A CA 2942776 A CA2942776 A CA 2942776A CA 2942776 C CA2942776 C CA 2942776C
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seq
sequence
cone
polynucleotide
expression
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French (fr)
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CA2942776A1 (en
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Jay Neitz
Maureen Neitz
Thomas W. Chalberg
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University of Washington
Adverum Biotechnologies Inc
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University of Washington
Adverum Biotechnologies Inc
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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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • 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
    • C12N2800/24—Vectors characterised by the absence of particular element, e.g. selectable marker, viral origin of replication
    • 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
    • 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
    • C12N2830/30—Vector systems having a special element relevant for transcription being an enhancer not forming part of the promoter region
    • 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
    • C12N2830/42—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)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • Virology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Mycology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Peptides Or Proteins (AREA)
CA2942776A 2014-03-17 2015-03-17 Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter Active CA2942776C (en)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201461954330P 2014-03-17 2014-03-17
US61/954,330 2014-03-17
US201562127185P 2015-03-02 2015-03-02
US62/127,185 2015-03-02
PCT/US2015/021087 WO2015142941A1 (en) 2014-03-17 2015-03-17 Compositions and methods for enhanced gene expression in cone cells

Publications (2)

Publication Number Publication Date
CA2942776A1 CA2942776A1 (en) 2015-09-24
CA2942776C true CA2942776C (en) 2023-01-24

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CA2942776A Active CA2942776C (en) 2014-03-17 2015-03-17 Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter

Country Status (11)

Country Link
US (3) US10000741B2 (enExample)
EP (2) EP3800191B1 (enExample)
JP (4) JP6689249B2 (enExample)
KR (2) KR102288849B1 (enExample)
CN (2) CN112626125A (enExample)
AU (3) AU2015231439B2 (enExample)
CA (1) CA2942776C (enExample)
ES (2) ES3042252T3 (enExample)
IL (2) IL259321B2 (enExample)
SG (2) SG11201607738WA (enExample)
WO (1) WO2015142941A1 (enExample)

Families Citing this family (67)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2011034947A2 (en) 2009-09-15 2011-03-24 University Of Washington Reagents and methods for modulating cone photoreceptor activity
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
TWI702955B (zh) 2012-05-15 2020-09-01 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒（ａａｖ）ｓｆｌｔ-１治療老年性黃斑部退化（ａｍｄ）
CN105247044B (zh) 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
WO2015142941A1 (en) 2014-03-17 2015-09-24 Avalanche Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
US11021519B2 (en) * 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
JP6969798B2 (ja) * 2015-12-03 2021-11-24 フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ ＳｙｎＰ１６０、桿体光受容器における遺伝子の特異的発現のためのプロモーター
CA3006952A1 (en) 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp161, a promoter for the specific expression of genes in rod photoreceptors
EP3795180B1 (en) 2015-12-14 2025-11-05 The Trustees of The University of Pennsylvania Gene therapy for ocular disorders
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
CN107287239B (zh) * 2016-04-11 2020-09-22 厦门继景生物技术有限责任公司 一种用于视网膜色素变性的基因治疗载体及药物
EP3448437B1 (en) * 2016-04-29 2021-12-29 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
WO2017205739A1 (en) 2016-05-26 2017-11-30 University Of Iowa Research Foundation cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1
CA3027740A1 (en) * 2016-06-16 2017-12-21 Adverum Biotechnologies, Inc. Compositions and methods for reducing ocular neovascularization
KR102526506B1 (ko) * 2016-07-08 2023-05-03 더 트러스티스 오브 더 유니버시티 오브 펜실베니아 Rdh12가 연루된 장애 및 질환의 치료를 위한 방법 및 조성물
KR20230039779A (ko) 2016-07-29 2023-03-21 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 갖는 아데노-관련된 바이러스 비리온 및 이의 사용 방법
US11192925B2 (en) 2016-10-19 2021-12-07 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
WO2018134168A1 (en) * 2017-01-17 2018-07-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors
EP4653536A2 (en) * 2017-03-17 2025-11-26 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
AU2018266381A1 (en) * 2017-05-08 2019-11-14 Lambdavision Incorporated A method for stimulating retinal cells and treating vision loss
EP3638316A4 (en) 2017-06-14 2021-03-24 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR EYE DISEASES
KR102766238B1 (ko) 2017-06-30 2025-02-13 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 변이체 캡시드를 보유한 아데노-부속 바이러스 비리온 및 이의 사용 방법
BR112020003571A2 (pt) 2017-08-28 2020-08-25 The Regents Of The University Of California variantes capsidiais de vírus adenoassociado e métodos de uso das mesmas
MX2020003176A (es) 2017-09-20 2020-08-20 4D Molecular Therapeutics Inc Variantes de capsides de virus adenoasociados y metodos de uso de estas.
EP3697448A1 (en) * 2017-10-20 2020-08-26 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant aav9-derived vector
WO2019094904A1 (en) * 2017-11-13 2019-05-16 The Regents Of The University Of California Compositions and methods for enhancing visual function
WO2019113225A1 (en) * 2017-12-05 2019-06-13 University Of Washington Compositions and methods for enhancing functional expression of therapeutic genes in photoreceptors
GB201800546D0 (en) * 2018-01-12 2018-02-28 Ucl Business Plc Treatment
WO2019169291A1 (en) 2018-03-02 2019-09-06 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Use of il-34 to treat retinal inflammation and neurodegeneration
CN109097364B (zh) * 2018-09-03 2022-02-11 深圳广三系农业科技有限公司 一种植物胚乳特异性表达启动子pOsEnS100的鉴定和应用
WO2020123862A1 (en) 2018-12-12 2020-06-18 Tesseract Health, Inc, Optical apparatus and associated devices for biometric identification and health status determination
WO2020167770A1 (en) * 2019-02-11 2020-08-20 President And Fellows Of Harvard College Txnip and ldhb compositions and methods for the treatment of degenerative ocular diseases
WO2020168111A1 (en) 2019-02-15 2020-08-20 Exhaura, Ltd. Dual leucine zipper kinase inhibitors for gene therapy
KR20220047538A (ko) * 2019-04-15 2022-04-18 유니버시티 오브 아이오와 리써치 파운데이션 트랜스진 발현을 위한 방법 및 조성물
US12516097B2 (en) 2019-04-19 2026-01-06 University Of Massachusetts Gene therapies for Stargardt disease (ABCA4)
US11737665B2 (en) 2019-06-21 2023-08-29 Tesseract Health, Inc. Multi-modal eye imaging with shared optical path
WO2021062012A1 (en) 2019-09-25 2021-04-01 Emory University Use of klk10 and engineered derivatizations thereof
EP4045644A4 (en) * 2019-10-17 2023-11-29 The Johns Hopkins University COMPOSITIONS AND METHODS OF USING ALTERNATIVE SPLICING TO REGULATE GENE THERAPY SPECIFICITY
AU2021208631A1 (en) 2020-01-17 2022-07-07 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Gene therapy for treatment of CRX-autosomal dominant retinopathies
MX2022011177A (es) * 2020-03-11 2022-12-13 Massachusetts Eye & Ear Infirmary Terapia genica para la degeneracion de la retina asociada a nmnat1.
WO2021204407A1 (en) 2020-04-10 2021-10-14 Sorbonne Université G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd)
WO2021222094A1 (en) 2020-04-27 2021-11-04 4D Molecular Therapeutics Inc. Codon optimized gla genes and uses thereof
CN111429547B (zh) * 2020-04-27 2023-05-30 中国人民解放军空军工程大学 一种基于假同色搜索的异常色觉测试图合成方法
WO2022020548A2 (en) * 2020-07-23 2022-01-27 President And Fellows Of Harvard College Variant txnip compositions and methods of use thereof for the treatment of degenerative ocular diseases
JP2023535956A (ja) * 2020-07-29 2023-08-22 ユニバーシティ オブ フロリダ リサーチ ファンデーション インコーポレーティッド 改善されたｘ連鎖性網膜分離症のａａｖ媒介療法
CN112062827B (zh) * 2020-09-16 2022-10-21 中国人民解放军军事科学院军事医学研究院 Cep55蛋白在调控纤毛去组装和制备纤毛相关疾病模型中的应用
GB202102832D0 (en) * 2021-02-26 2021-04-14 Ip2Ipo Innovations Ltd Retroviral vectors
US12385064B2 (en) * 2021-03-26 2025-08-12 Adverum Biotechnologies, Inc. Intravitreal dosing for delivery of polynucleotides to retinal cones
GB202104611D0 (en) * 2021-03-31 2021-05-12 Univ Oxford Innovation Ltd Gene therapy for retinal disease
US20230049217A1 (en) * 2021-05-21 2023-02-16 Novartis Ag Compositions and methods for enhancing visual function
JP2024520558A (ja) 2021-05-26 2024-05-24 スパーリングヴィジョン Ｇタンパク質ゲート－ｋ＋チャネルを介した桿体－錐体ジストロフィー（ｒｃｄ）における光感受性の増強
CN113408798B (zh) * 2021-06-14 2022-03-29 华中师范大学 一种面向色觉异常人群的无障碍vr教学资源色彩优化方法
KR102876040B1 (ko) 2021-07-30 2025-10-23 주식회사 엘지에너지솔루션 이차 전지용 전극, 이의 제조방법, 및 상기 전극을 포함하는 이차 전지
EP4399314A4 (en) * 2021-09-06 2025-09-10 Huidagene Therapeutics Singapore Pte Ltd TREATMENT OF EYE DISEASES AND DISORDERS ASSOCIATED WITH RPE65
EP4163296A1 (en) 2021-10-11 2023-04-12 Sparingvision G-protein-gated-k+ channel-mediated enhancements in light sensitivity in patients with rod-cone dystrophy (rcd)
WO2023122804A1 (en) * 2021-12-24 2023-06-29 The Trustees Of The University Of Pennsylvania Compositions and methods comprising a cardiac-specific promoter
WO2023205758A2 (en) * 2022-04-22 2023-10-26 West Virginia University Board of Governors on behalf of West Virginia University Raav-cone opsin compositions and methods for treating blue cone monochromacy and color blindness
CN114848850B (zh) * 2022-04-28 2023-12-15 武汉中眸生物科技有限责任公司 Rs1基因在制备xlrs治疗剂中的应用及治疗剂
CA3257440A1 (en) * 2022-07-06 2024-01-11 Adverum Biotechnologies, Inc. COMPOSITIONS AND METHODS FOR THE TREATMENT OF ACHROMOTOPSY
WO2024073564A2 (en) * 2022-09-30 2024-04-04 Senti Biosciences, Inc. Cell-type specific regulatory elements for photoreceptors
US20240207451A1 (en) 2022-10-20 2024-06-27 Sparingvision G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd)
CN118791624A (zh) * 2023-04-11 2024-10-18 苏州星明优健生物技术有限公司 用于治疗视网膜神经退行性疾病的新型gpcr嵌合视蛋白
WO2025022144A1 (en) * 2023-07-26 2025-01-30 Sorbonne Universite Chimeric promoter for targeted expression in aii amacrine cells
WO2025113432A1 (zh) * 2023-11-27 2025-06-05 北京因诺惟康医药科技有限公司 一种用于治疗x染色体连锁视网膜劈裂症的基因药物
CN117625619B (zh) * 2023-12-05 2024-11-12 云舟生物科技(广州)股份有限公司 核酸分子及其作为特异启动子的应用
CN118064572B (zh) * 2024-03-15 2025-07-01 温州谱希基因科技有限公司 一种用于红绿色觉异常诊断的引物探针组合和试剂盒及其应用
CN118562953A (zh) * 2024-06-21 2024-08-30 复旦大学附属眼耳鼻喉科医院 检测opn1lw与opn1mw基因单倍体型的试剂在近视诊断试剂盒中的应用及产品

Family Cites Families (194)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4522811A (en) 1982-07-08 1985-06-11 Syntex (U.S.A.) Inc. Serial injection of muramyldipeptides and liposomes enhances the anti-infective activity of muramyldipeptides
US4874237A (en) 1987-05-07 1989-10-17 Lions Eye Inst. Of Western Australia Electroretinogram apparatus
JP2755817B2 (ja) 1988-11-14 1998-05-25 アメリカ合衆国 パルボウイルスキャプシド
FR2643252B1 (fr) 1989-02-21 1991-06-07 Technomed Int Sa Appareil de destruction selective de cellules incluant les tissus mous et les os a l'interieur du corps d'un etre vivant par implosion de bulles de gaz
US5112946A (en) 1989-07-06 1992-05-12 Repligen Corporation Modified pf4 compositions and methods of use
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6287815B1 (en) 1989-09-14 2001-09-11 Rijksuniversiteit Te Leiden Human parvovirus B19 proteins and virus-like particles, their production and their use in diagnostic assays and vaccines
ATE159548T1 (de) 1990-11-13 1997-11-15 Immunex Corp Bifunktionelle wählbare fusionsgene
US5383917A (en) 1991-07-05 1995-01-24 Jawahar M. Desai Device and method for multi-phase radio-frequency ablation
GB9206016D0 (en) 1992-03-19 1992-04-29 Sandoz Ltd Improvements in or relating to organic compounds
DK0694042T3 (da) 1993-03-25 2005-02-14 Merck & Co Inc Inhibitor af vaskulær endothelcellevækstfaktor
AU6953394A (en) 1993-05-21 1994-12-20 Targeted Genetics Corporation Bifunctional selectable fusion genes based on the cytosine deaminase (cd) gene
US5814618A (en) 1993-06-14 1998-09-29 Basf Aktiengesellschaft Methods for regulating gene expression
AU1925195A (en) 1994-02-22 1995-09-04 Dana-Farber Cancer Institute Nucleic acid delivery system, method of synthesis and uses thereof
US6551618B2 (en) 1994-03-15 2003-04-22 University Of Birmingham Compositions and methods for delivery of agents for neuronal regeneration and survival
FR2718150B1 (fr) 1994-03-29 1996-04-26 Rhone Poulenc Rorer Sa Virus recombinants, préparation et utilisation en thérapie génique.
US5639725A (en) 1994-04-26 1997-06-17 Children's Hospital Medical Center Corp. Angiostatin protein
US5527533A (en) 1994-10-27 1996-06-18 Board Of Trustees Of The University Of Illinois Method of retarding and ameliorating central nervous system and eye damage
US20020168342A1 (en) 1994-11-03 2002-11-14 Cell Genesys, Inc. Novel adenoviral vectors, packaging cell lines, recombinant adenoviruses and methods
US6440425B1 (en) 1995-05-01 2002-08-27 Aventis Pasteur Limited High molecular weight major outer membrane protein of moraxella
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6001650A (en) 1995-08-03 1999-12-14 Avigen, Inc. High-efficiency wild-type-free AAV helper functions
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
US5641749A (en) 1995-11-29 1997-06-24 Amgen Inc. Method for treating retinal ganglion cell injury using glial cell line-derived neurothrophic factor (GDNF) protein product
US6096548A (en) 1996-03-25 2000-08-01 Maxygen, Inc. Method for directing evolution of a virus
AU2678097A (en) 1996-04-16 1997-11-07 Immusol Incorporated Targeted viral vectors
EP1003530A4 (en) 1996-08-20 2001-10-04 Univ California EYE TREATMENT USING SYNTHETIC THYROID HORMONE COMPOSITIONS
EP0928203B1 (en) 1996-09-24 2006-10-25 Merck & Co., Inc. Compounds for the inhibition of angiogenesis by gene therapy
FR2756297B1 (fr) 1996-11-22 1999-01-08 Centre Nat Rech Scient Procede de production de virus recombinants
US6153436A (en) 1997-01-10 2000-11-28 The Board Of Trustees Of The University Of Arkansas Method of gene delivery using wildtype adeno associated viral (AAV) vectors with insertions
EP1007637B1 (en) 1997-04-14 2004-06-30 Cell Genesys, Inc. Methods for increasing the efficiency of recombinant aav product
AU7383298A (en) 1997-05-13 1998-12-08 Regents Of The University Of California, The Novel antiangiogenic peptide agents and their therapeutic and diagnostic use
US6710036B2 (en) 1997-07-25 2004-03-23 Avigen, Inc. Induction of immune response to antigens expressed by recombinant adeno-associated virus
US6458157B1 (en) 1997-08-04 2002-10-01 Suaning Gregg Joergen Retinal stimulator
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
JPH11100327A (ja) 1997-09-26 1999-04-13 Toray Ind Inc 網膜変性症治療剤
AU9673998A (en) 1997-10-01 1999-04-23 G.D. Searle & Co. Fusion proteins comprising an angiostatin moiety and their use in anti-tumor treatment
WO1999023107A1 (en) 1997-10-31 1999-05-14 Maxygen, Incorporated Modification of virus tropism and host range by viral genome shuffling
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
WO1999036511A2 (en) 1998-01-16 1999-07-22 Chiron Corporation Feline immunodeficiency virus gene therapy vectors
US6551795B1 (en) 1998-02-18 2003-04-22 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to pseudomonas aeruginosa for diagnostics and therapeutics
ES2186340T3 (es) 1998-03-12 2003-05-01 Genentech Inc Utilizacion de polipeptidos fgf-5 para la prevencion de la muerte de neuronas retinales y para el tratamiento de enfermedades oculares.
DE19827457C1 (de) 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
US6593133B1 (en) 1998-07-06 2003-07-15 Nsgene A/S Neurotrophic factors
IL141403A0 (en) 1998-08-28 2002-03-10 Univ Duke Deleted adenoviruses and methods of making and administering the same
CN1159445C (zh) 1998-09-11 2004-07-28 加利福尼亚大学董事会 能在心脏中组织特异性表达的重组腺病毒
CA2346262A1 (en) 1998-09-17 2000-03-23 University Of Florida Methods for treatment of degenerative retinal diseases
US20040234505A1 (en) 1998-09-23 2004-11-25 Stuart Naylor Polynucleotide constructs and uses thereof
AU780231B2 (en) 1998-11-10 2005-03-10 University Of North Carolina At Chapel Hill, The Virus vectors and methods of making and administering the same
US6943153B1 (en) 1999-03-15 2005-09-13 The Regents Of The University Of California Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
JP2002539176A (ja) 1999-03-15 2002-11-19 カイロン コーポレイション 眼の疾患を処置または予防するための組換え遺伝子送達ベクターの使用
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
US7306799B2 (en) 1999-06-08 2007-12-11 Regeneron Pharmaceuticals, Inc. Use of VEGF inhibitors for treatment of eye disorders
US7087411B2 (en) 1999-06-08 2006-08-08 Regeneron Pharmaceuticals, Inc. Fusion protein capable of binding VEGF
US7070959B1 (en) 1999-06-08 2006-07-04 Regeneron Pharmaceuticals, Inc. Modified chimeric polypeptides with improved pharmacokinetic properties
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
DE19933719A1 (de) 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
US8232081B2 (en) 1999-09-21 2012-07-31 Basf Se Methods and microorganisms for production of panto-compounds
US6656136B1 (en) 1999-10-25 2003-12-02 Therus Corporation Use of focused ultrasound for vascular sealing
WO2002081628A2 (en) 2001-04-05 2002-10-17 Ribozyme Pharmaceuticals, Incorporated Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
EP1274339B1 (en) 2000-04-17 2011-06-08 The University of Sydney Method for objective electrophysiological assessment of visual function
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
AU2002236988A1 (en) 2001-02-06 2002-08-19 Novartis Ag Photodynamic therapy of occult choroidal neovascularization linked to age-related macular degeneration
WO2002076355A2 (de) 2001-03-27 2002-10-03 Wavelight Laser Technologie Ag Verfahren und vorrichtung zur bearbeitung und diagnose von augengewebe
WO2002082904A2 (en) 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
JP2002363107A (ja) 2001-06-04 2002-12-18 Noriyuki Azuma 色覚不全動物の色覚復元方法
EP1412493B1 (en) 2001-08-02 2011-10-05 Institut Clayton De La Recherche Methods and compositions relating to improved lentiviral vector production systems
US20030129203A1 (en) 2001-08-27 2003-07-10 Nautilus Biotech S.A. Mutant recombinant adeno-associated viruses
US7647184B2 (en) 2001-08-27 2010-01-12 Hanall Pharmaceuticals, Co. Ltd High throughput directed evolution by rational mutagenesis
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
IL161827A0 (en) 2001-11-13 2005-11-20 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novelsequences identified thereby
US20050106558A1 (en) 2001-12-21 2005-05-19 Luca Perabo Library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
WO2003080648A2 (en) 2002-03-20 2003-10-02 University Of Florida Research Foundation, Inc. Raav vector compositions and methods for the treatment of choroidal neovascularization
US20060292117A1 (en) 2002-04-17 2006-12-28 Loiler Scott A Improved rAAv vectors
WO2003092594A2 (en) 2002-04-30 2003-11-13 Duke University Adeno-associated viral vectors and methods for their production from hybrid adenovirus and for their use
WO2003093436A2 (en) 2002-04-30 2003-11-13 University Of Florida Treatment for phenylketonuria
US7254489B2 (en) 2002-05-31 2007-08-07 Microsoft Corporation Systems, methods and apparatus for reconstructing phylogentic trees
US8304233B2 (en) 2002-06-04 2012-11-06 Poetic Genetics, Llc Methods of unidirectional, site-specific integration into a genome, compositions and kits for practicing the same
US7220577B2 (en) 2002-08-28 2007-05-22 University Of Florida Research Foundation, Inc. Modified AAV
GB0220467D0 (en) 2002-09-03 2002-10-09 Oxford Biomedica Ltd Composition
JP2006519026A (ja) 2003-03-04 2006-08-24 独立行政法人産業技術総合研究所 標的物質の細胞導入効率を上昇させるための組成物および方法
WO2004083441A2 (en) 2003-03-19 2004-09-30 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Random peptide library displayed on aav vectors
WO2004108922A2 (en) 2003-04-25 2004-12-16 The Trustees Of The University Of Pennsylvania Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
DK2657247T3 (en) 2003-06-19 2017-07-10 Genzyme Corp AAV virions with reduced immunoreactivity and applications thereof
EP1644038A2 (en) 2003-06-23 2006-04-12 A & G Pharmaceuticals, Inc. Compositions and methods for restoring sensitivity of tumor cells to antitumor therapy and inducing apoptosis
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US20050148069A1 (en) 2003-09-24 2005-07-07 Gage Fred H. IGF-1 instructs multipotent adult CNS neural stem cells to an oligodendroglial lineage
EP3211085B1 (en) 2003-09-30 2021-03-31 The Trustees of The University of Pennsylvania Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
NZ596663A (en) 2004-10-21 2013-07-26 Genentech Inc Use of vegf antagonists in intraocular neovascular disease treatment
ES2384391T3 (es) 2004-11-08 2012-07-04 Chromagenics B.V. Selección de células hospedantes que expresan proteína a altos niveles
AU2005316476A1 (en) 2004-12-15 2006-06-22 University Of Florida Research Foundation, Inc. Chimeric vectors
US20100172871A1 (en) 2005-02-17 2010-07-08 Flannery John G Muller Cell Specific Gene Therapy
US7922670B2 (en) 2005-02-24 2011-04-12 Warren Jones System and method for quantifying and mapping visual salience
CN104293835B (zh) 2005-04-07 2017-07-04 宾夕法尼亚大学托管会 增强腺相关病毒载体功能的方法
US20060234347A1 (en) 2005-04-13 2006-10-19 Harding Thomas C Targeting multiple angiogenic pathways for cancer therapy using soluble tyrosine kinase receptors
WO2007046703A2 (en) 2005-10-20 2007-04-26 Amsterdam Molecular Therapeutics B.V. Improved aav vectors produced in insect cells
EP1981548A4 (en) 2006-01-20 2010-03-24 Univ North Carolina REINFORCED PRODUCTION OF INFECTIOUS PARVOVIRUS VECTORS IN INSECT CELLS
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
US20070190028A1 (en) 2006-02-13 2007-08-16 Jihong Qu Method and apparatus for heat or electromagnetic control of gene expression
US8118752B2 (en) 2006-02-16 2012-02-21 The Board Of Trustees Of The University Of Illinois Apparatus and methods for mapping retinal function
US7384145B2 (en) 2006-02-16 2008-06-10 The Board Of Trustees Of The University Of Illinois Mapping retinal function using corneal electrode array
WO2007120542A2 (en) 2006-03-30 2007-10-25 The Board Of Trustees Of The Leland Stanford Junior University Aav capsid library and aav capsid proteins
US8216575B2 (en) 2006-03-31 2012-07-10 Chengdu Kanghong Biotechnologies Co., Ltd. Inhibition of neovascularization with a soluble chimeric protein comprising VEGF FLT-1 and KDR domains
AU2007247426B2 (en) 2006-05-04 2012-03-08 Fondation Ophthalmologique Adolf De Rothschild Combination comprising a VEGF inhibitor and a serine protease for treating neovascular diseases
CA2685900A1 (en) 2006-05-04 2007-11-15 Wayne State University Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
PT2029746E (pt) 2006-06-12 2012-10-15 Exegenics Inc D B A Opko Health Inc Composições e métodos para a inibição do sirna de angiogenese
GB0612096D0 (en) 2006-06-19 2006-07-26 Greater Glasgow Nhs Board Functional imaging of the retina
WO2007148971A2 (en) 2006-06-21 2007-12-27 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
US20100008170A1 (en) 2006-06-27 2010-01-14 Shinya Sato Semiconductor tester and testing method of semiconductor memory
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
CN1966082B (zh) 2006-11-03 2010-06-30 许瑞安 一种防治结直肠癌的基因药物及其制备方法和用途
WO2008098310A1 (en) 2007-02-16 2008-08-21 Objectivision Limited Stimulus method for multifocal visual evoked potential
US9725485B2 (en) 2012-05-15 2017-08-08 University Of Florida Research Foundation, Inc. AAV vectors with high transduction efficiency and uses thereof for gene therapy
NZ579985A (en) 2007-04-13 2012-02-24 Catalyst Biosciences Inc Modified factor vii polypetides and uses thereof
EP1985708B1 (en) 2007-04-27 2015-04-15 Universität Rostock Selective targeting of viruses to neural precursor cells
EP1995309A1 (en) 2007-05-21 2008-11-26 Vivalis Recombinant protein production in avian EBx® cells
US10696983B2 (en) 2007-05-30 2020-06-30 The Trustees Of The University Of Pennsylvania Method for transducing cells with primary cilia
US8540369B2 (en) 2007-08-16 2013-09-24 The Research Foundation Of State University Of New York Led variable light source
US8518037B2 (en) 2007-10-30 2013-08-27 Boston Scientific Scimed, Inc. Radiofrequency ablation device
US20110053274A1 (en) 2007-11-30 2011-03-03 Scarab Genomics Llc Lac expression system
EA020969B1 (ru) 2008-02-19 2015-03-31 ЮНИКЬЮРЕ АйПи Б.В. ОПТИМИЗАЦИЯ ЭКСПРЕССИИ ПАРВОВИРУСНЫХ БЕЛКОВ Rep И Cap В КЛЕТКАХ НАСЕКОМЫХ
WO2009105669A2 (en) 2008-02-20 2009-08-27 Genzyme Corporation Angiogenesis inhibition
WO2009105690A2 (en) 2008-02-21 2009-08-27 Targeted Genetics Corporation Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
ITFI20080081A1 (it) 2008-04-18 2009-10-19 Strumenti Oftalmici C S O S R Procedimento e sistema per la registrazione di risposte elettrofunzionali erg, perg e vep multifocali in tempo reale
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
CN102159713A (zh) 2008-05-20 2011-08-17 Eos神经科学公司 用于递送光敏蛋白的载体和使用方法
ES2330826B1 (es) 2008-06-04 2010-07-26 Proyecto De Biomedicina Cima, S.L. Sistema para empaquetamiento de adenovirus de alta capacidad.
EP2297185A1 (en) 2008-06-17 2011-03-23 Amsterdam Molecular Therapeutics (AMT) B.V. Parvoviral capsid with incorporated gly-ala repeat region
JP5491511B2 (ja) 2008-10-07 2014-05-14 ブラッコ・シュイス・ソシエテ・アノニム 抗ポリマー抗体およびそれと結合するリポソームまたは微小胞を含むターゲッティング構築物
WO2010093784A2 (en) 2009-02-11 2010-08-19 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
ES2742180T3 (es) 2009-03-04 2020-02-13 Deutsches Krebsforsch Proteína activadora del ensamblaje (AAP) y su uso para la fabricación de partículas de parvovirus que consisten esencialmente en VP3
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
CN101532024A (zh) 2009-04-30 2009-09-16 许瑞安 一种用于基因治疗的新型细胞特异性内含microRNA结合序列的基因HAAVmir
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
TWI466158B (zh) 2009-07-03 2014-12-21 Univ Lunghwa Sci & Technology 電漿測量裝置、電漿系統及測量電漿特性之方法
EP2287323A1 (en) 2009-07-31 2011-02-23 Association Institut de Myologie Widespread gene delivery to the retina using systemic administration of AAV vectors
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
WO2011034947A2 (en) 2009-09-15 2011-03-24 University Of Washington Reagents and methods for modulating cone photoreceptor activity
US20110270256A1 (en) 2009-09-24 2011-11-03 Medicinelodge, Inc. Dba Imds Co-Innovation Surgical rasp with radiofrequency ablation
WO2011088081A1 (en) 2010-01-12 2011-07-21 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
US9228174B2 (en) 2010-03-11 2016-01-05 Uniqure Ip B.V. Mutated rep encoding sequences for use in AAV production
WO2011117258A2 (en) 2010-03-22 2011-09-29 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
MX342858B (es) 2010-03-29 2016-10-13 The Trustees Of The Univ Of Pennsylvania * Sistema de ablacion transgenica inducida farmacologicamente.
WO2011122950A1 (en) 2010-04-01 2011-10-06 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Monomeric duplex aav vectors
US9102718B2 (en) 2010-04-30 2015-08-11 Lpath, Inc. Anti-S1P antibody treatment of patients with ocular disease
CN201704771U (zh) 2010-04-30 2011-01-12 陈福环 无水箱节水型抽水马桶
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US20110052678A1 (en) 2010-11-05 2011-03-03 Shantha Totada R Method for treating age related macular degeneration
DK2640743T3 (en) 2010-11-16 2017-01-23 Excelimmune Inc PROCEDURES FOR THE PREPARATION OF RECOMBINANT PROTEINS
EP2661494B1 (en) 2011-01-07 2019-06-12 Applied Genetic Technologies Corporation Promoters, expression cassettes and vectors for use treating achromatopsia and other diseases
CN105755044A (zh) 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
EP2748185A1 (en) 2011-08-24 2014-07-02 The Board of Trustees of The Leland Stanford Junior University New aav capsid proteins for nucleic acid transfer
WO2013063601A1 (en) 2011-10-28 2013-05-02 University Of Florida Research Foundation, Inc. Chimeric promoter for cone photoreceptor targeted gene therapy
EP2847337A4 (en) 2012-05-09 2016-04-27 Univ Oregon Health & Science ADENO ASSOCIATED VIRUS PLASMIDS AND VECTORS
TWI702955B (zh) 2012-05-15 2020-09-01 澳大利亞商艾佛蘭屈澳洲私營有限公司 使用腺相關病毒（ａａｖ）ｓｆｌｔ-１治療老年性黃斑部退化（ａｍｄ）
US20150111275A1 (en) 2012-06-11 2015-04-23 Daniel V. Palanker Optical regulation of gene expression in the retina
WO2014103957A1 (ja) 2012-12-25 2014-07-03 タカラバイオ株式会社 Ａａｖ変異体
ES3053688T3 (en) * 2013-05-16 2026-01-23 Beacon Therapeutics Usa Inc Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
AU2014273085B2 (en) 2013-05-29 2020-10-22 Cellectis New compact scaffold of Cas9 in the type II CRISPR system
CN105247044B (zh) 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
ES2716615T3 (es) 2013-06-28 2019-06-13 Inst Nat Sante Rech Med Métodos para expresar un polinucleótido de interés en la retina de un sujeto
US20150025939A1 (en) 2013-07-18 2015-01-22 Somnath Chatterjee Company Centric Social Media Platfonn for Content Sharing aud Tracking
WO2015038958A1 (en) 2013-09-13 2015-03-19 California Institute Of Technology Selective recovery
EP3049527A4 (en) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
EP3459965B1 (en) 2013-10-11 2020-12-02 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
CA2926853C (en) 2013-10-18 2022-04-26 Regeneron Pharmaceuticals, Inc. Methods and compositions comprising a combination of a vegf antagonist and an anti-ctla-4 antibody
EP3113787B1 (en) 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
WO2015142941A1 (en) 2014-03-17 2015-09-24 Avalanche Biotechnologies, Inc. Compositions and methods for enhanced gene expression in cone cells
EA034355B1 (ru) 2014-05-02 2020-01-30 Джензим Корпорейшн Векторы на основе aav для генной терапии в сетчатке и cns
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
US11021519B2 (en) 2015-03-02 2021-06-01 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
JP6812368B2 (ja) 2015-03-06 2021-01-13 マサチューセッツ アイ アンド イヤー インファーマリー Ｐｒｐｆ３１遺伝子における変異により引き起こされる遺伝性網膜変性に対する遺伝子増強療法
EP3277819B1 (en) 2015-03-24 2021-03-03 The Regents of The University of California Adeno-associated virus variants and methods of use thereof
WO2017023724A1 (en) 2015-07-31 2017-02-09 Voyager Therapeutics, Inc. Compositions and methods for the treatment of aadc deficiency
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
EP3448437B1 (en) 2016-04-29 2021-12-29 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
AU2017259968A1 (en) 2016-05-03 2018-11-15 Adverum Biotechnologies, Inc. Organotypic pig retina culture system
RU2725286C2 (ru) 2016-05-13 2020-06-30 4Д Молекьюлар Терапьютикс Инк. Варианты капсидов аденоассоциированного вируса и способы их применения
CA3027740A1 (en) 2016-06-16 2017-12-21 Adverum Biotechnologies, Inc. Compositions and methods for reducing ocular neovascularization
LT3471780T (lt) 2016-06-16 2021-01-25 Adverum Biotechnologies, Inc. Amd gydymas, naudojant aav2 variantą su afliberceptu
US11192925B2 (en) 2016-10-19 2021-12-07 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
CN110650733A (zh) 2017-02-28 2020-01-03 阿德夫拉姆生物技术股份有限公司 经过修饰的aav衣壳和其用途
EP4653536A2 (en) 2017-03-17 2025-11-26 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
BR112020003571A2 (pt) 2017-08-28 2020-08-25 The Regents Of The University Of California variantes capsidiais de vírus adenoassociado e métodos de uso das mesmas

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