SG11201607738WA - Compositions and methods for enhanced gene expression in cone cells - Google Patents

Compositions and methods for enhanced gene expression in cone cells

Info

Publication number
SG11201607738WA
SG11201607738WA SG11201607738WA SG11201607738WA SG11201607738WA SG 11201607738W A SG11201607738W A SG 11201607738WA SG 11201607738W A SG11201607738W A SG 11201607738WA SG 11201607738W A SG11201607738W A SG 11201607738WA SG 11201607738W A SG11201607738W A SG 11201607738WA
Authority
SG
Singapore
Prior art keywords
compositions
methods
gene expression
cone cells
enhanced gene
Prior art date
Application number
SG11201607738WA
Other languages
English (en)
Inventor
Thomas W Chalberg
Jay Neitz
Maureen Neitz
Original Assignee
Adverum Biotechnologies Inc
Univ Washington
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Adverum Biotechnologies Inc, Univ Washington filed Critical Adverum Biotechnologies Inc
Publication of SG11201607738WA publication Critical patent/SG11201607738WA/en

Links

Classifications

    • 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
    • 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
    • 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
    • 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
    • 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

Landscapes

  • 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)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Microbiology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (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)
  • Peptides Or Proteins (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Gastroenterology & Hepatology (AREA)
SG11201607738WA 2014-03-17 2015-03-17 Compositions and methods for enhanced gene expression in cone cells SG11201607738WA (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201461954330P 2014-03-17 2014-03-17
US201562127185P 2015-03-02 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 (1)

Publication Number Publication Date
SG11201607738WA true SG11201607738WA (en) 2016-10-28

Family

ID=54068212

Family Applications (2)

Application Number Title Priority Date Filing Date
SG10201810150UA SG10201810150UA (en) 2014-03-17 2015-03-17 Compositions and methods for enhanced gene expression in cone cells
SG11201607738WA SG11201607738WA (en) 2014-03-17 2015-03-17 Compositions and methods for enhanced gene expression in cone cells

Family Applications Before (1)

Application Number Title Priority Date Filing Date
SG10201810150UA SG10201810150UA (en) 2014-03-17 2015-03-17 Compositions and methods for enhanced gene expression in cone cells

Country Status (11)

Country Link
US (3) US10000741B2 (enExample)
EP (2) EP3800191B1 (enExample)
JP (4) JP6689249B2 (enExample)
KR (2) KR102288849B1 (enExample)
CN (2) CN106414474B (enExample)
AU (3) AU2015231439B2 (enExample)
CA (1) CA2942776C (enExample)
ES (2) ES2822551T3 (enExample)
IL (2) IL259321B2 (enExample)
SG (2) SG10201810150UA (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
US20120172419A1 (en) 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
DK2699270T3 (en) 2011-04-22 2017-10-02 Univ California ADENO-ASSOCIATED VIRUS VIRUSES WITH VARIANT CAPSID AND METHODS FOR USING IT
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）
ES2897508T3 (es) 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
JP6689249B2 (ja) 2014-03-17 2020-05-20 アドヴェラム バイオテクノロジーズ， インコーポレイテッド 錐体細胞における増強された遺伝子発現のための組成物および方法
AU2016226289B2 (en) 2015-03-02 2021-04-29 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
ES2900486T3 (es) * 2015-12-03 2022-03-17 Friedrich Miescher Institute For Biomedical Res SynP160, un promotor para la expresión específica de genes en los fotorreceptores de los bastones
EP3383439B1 (en) * 2015-12-03 2019-09-25 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
WO2017190125A1 (en) * 2016-04-29 2017-11-02 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
US12173305B2 (en) 2016-05-26 2024-12-24 University Of Iowa Research Foundation cis and trans requirements for terminal resolution of human bocavirus 1
EP4427808A3 (en) * 2016-06-16 2024-10-30 Adverum Biotechnologies, Inc. Compositions and methods for reducing ocular neovascularization
IL264070B2 (en) 2016-07-08 2023-03-01 Univ Pennsylvania Methods and preparations for the treatment of disorders and diseases involving rdh12
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
CA3040179A1 (en) 2016-10-19 2018-04-26 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
CA3054942A1 (en) 2017-03-17 2018-09-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
EP3618763A4 (en) * 2017-05-08 2021-02-17 Lambdavision Incorporated A method for stimulating retinal cells and treating vision loss
WO2018232149A1 (en) * 2017-06-14 2018-12-20 The Trustees Of The University Of Pennsylvania Gene therapy for ocular disorders
WO2019006182A1 (en) 2017-06-30 2019-01-03 The Regents Of The University Of California VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS AND METHODS OF USE THEREOF
CA3059995A1 (en) 2017-08-28 2019-03-07 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof
NZ763018A (en) * 2017-09-20 2023-02-24 4D Molecular Therapeutics Inc Adeno-associated virus variant capsids and methods of use thereof
US11723988B2 (en) 2017-10-20 2023-08-15 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
EP3709871A4 (en) 2017-11-13 2021-12-01 The Regents of The University of California COMPOSITIONS AND METHODS TO IMPROVE VISUAL FUNCTION
EP3720460A1 (en) * 2017-12-05 2020-10-14 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
CA3091810A1 (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
CA3121791A1 (en) 2018-12-12 2020-06-18 Tesseract Health, Inc. 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
WO2020214672A1 (en) * 2019-04-15 2020-10-22 University Of Iowa Research Foundation Methods and compositions for transgene expression
CN114126665B (zh) * 2019-04-19 2025-04-25 马萨诸塞大学 眼底黄色斑点症(abca4)的基因疗法
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
CA3154826A1 (en) * 2019-10-17 2021-04-22 Jonathan P. LING Compositions and methods for using alternative splicing to control specificity of gene therapy
CA3165922A1 (en) 2020-01-17 2021-07-22 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
US12257320B2 (en) 2020-03-11 2025-03-25 Massachusetts Eye And Ear Infirmary Gene therapy for NMNAT1-associated retinal degeneration
EP4132961A1 (en) 2020-04-10 2023-02-15 Sorbonne Université G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd)
IL297404A (en) 2020-04-27 2022-12-01 4D Molecular Therapeutics Inc Codon-optimized gla genes and their uses
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
EP4185701A4 (en) * 2020-07-23 2025-01-01 President and Fellows of Harvard College TXNIP VARIANT COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING DEGENERATIVE EYE DISEASES
CN116568338A (zh) * 2020-07-29 2023-08-08 佛罗里达州大学研究基金会 改进的aav介导的x连锁视网膜劈裂症治疗
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
UY39772A (es) * 2021-05-21 2023-01-31 Novartis Ag Composiciones y métodos para mejorar la función visual
WO2022248634A1 (en) 2021-05-26 2022-12-01 Sparingvision G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd)
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
US20250263460A1 (en) * 2022-04-22 2025-08-21 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
TW202421773A (zh) * 2022-09-30 2024-06-01 美商聖堤生物科技股份有限公司 用於光受體之細胞型特異性調控元件
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
WO1990005538A1 (en) 1988-11-14 1990-05-31 The United States Of America, As Represented By The Secretary, U.S. Department Of Commerce Parvovirus capsids
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
JP3150340B2 (ja) 1990-11-13 2001-03-26 イムネクス コーポレイション 二機能選択可能融合遺伝子
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
EP0694042B1 (en) 1993-03-25 2004-11-03 Merck & Co. Inc. Inhibitor of vascular endothelial cell growth factor
JPH09500783A (ja) 1993-05-21 1997-01-28 ターゲッティッド ジェネティクス コーポレイション シトシンデアミナーゼ（ｃｄ）遺伝子に基づく二機能性選択融合遺伝子
US5814618A (en) 1993-06-14 1998-09-29 Basf Aktiengesellschaft Methods for regulating gene expression
EP0745134A1 (en) 1994-02-22 1996-12-04 Danafarber 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
US6040183A (en) 1995-06-07 2000-03-21 University Of North Carloina At Chapel Hill Helper virus-free AAV production
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina 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
WO1997038723A1 (en) 1996-04-16 1997-10-23 Immusol Incorporated Targeted viral vectors
WO1998007435A1 (en) 1996-08-20 1998-02-26 The Regents Of The University Of California Eye treatments using synthetic thyroid hormone compositions
JP2001501471A (ja) 1996-09-24 2001-02-06 メルク エンド カンパニー インコーポレーテッド 血管新生の阻害のための遺伝子治療
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
DE69824859T2 (de) 1997-04-14 2005-08-04 Cell Genesys, Inc., Foster City Methoden zur erhöhung der effizienz rekombinanter aav produkte
WO1998051323A1 (en) 1997-05-13 1998-11-19 The Regents Of The University Of California 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
EP1705248A1 (en) 1997-10-01 2006-09-27 G.D. Searle LLC. Fusion proteins comprising an angiostatin moiety and their use in anti-tumour treatment
AU1449499A (en) 1997-10-31 1999-05-24 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
EP1045921A2 (en) 1998-01-16 2000-10-25 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
IL138033A0 (en) 1998-03-12 2001-10-31 Genentech Inc Method of preventing the death of retinal neurons and treating ocular diseases
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
JP2002528056A (ja) 1998-08-28 2002-09-03 デューク・ユニバーシティー ＩＶａ２、１００Ｋ及び／又は末端前タンパク質配列における欠失型アデノウイルス
ATE294874T1 (de) 1998-09-11 2005-05-15 Univ California Rekombinante adenovirus für gewebespezifische genexpression ins herz
AU775245B2 (en) 1998-09-17 2004-07-22 Catholic University Nijmegen Methods for treatment of degenerative retinal diseases
US20040234505A1 (en) 1998-09-23 2004-11-25 Stuart Naylor Polynucleotide constructs and uses thereof
DE69941905D1 (de) 1998-11-10 2010-02-25 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
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
WO2000054813A2 (en) 1999-03-15 2000-09-21 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
US7070959B1 (en) 1999-06-08 2006-07-04 Regeneron Pharmaceuticals, Inc. Modified chimeric polypeptides with improved pharmacokinetic properties
US7087411B2 (en) 1999-06-08 2006-08-08 Regeneron Pharmaceuticals, Inc. Fusion protein capable of binding VEGF
US7306799B2 (en) 1999-06-08 2007-12-11 Regeneron Pharmaceuticals, Inc. Use of VEGF inhibitors for treatment of eye disorders
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
WO2001034018A2 (en) 1999-10-25 2001-05-17 Therus Corporation Use of focused ultrasound for vascular sealing
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
JP4817582B2 (ja) 2000-04-17 2011-11-16 ザ・ユニバーシティー・オブ・シドニー 視覚機能の対物電気生理学的評価方法および装置
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
US8034803B2 (en) 2001-02-06 2011-10-11 Qlt Inc. Photodynamic therapy of occult age-related macular degeneration
EP1372552B1 (de) 2001-03-27 2017-03-01 WaveLight GmbH Vorrichtung zur bearbeitung und diagnose von augengewebe
EP1386004A4 (en) 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
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
DK1412493T3 (da) 2001-08-02 2012-01-09 Inst Clayton De La Rech Fremgangsmåder og sammensætninger der angår forbedrede lentiviral-vektor.-produktionsystemer
US7647184B2 (en) 2001-08-27 2010-01-12 Hanall Pharmaceuticals, Co. Ltd High throughput directed evolution by rational mutagenesis
US20030129203A1 (en) 2001-08-27 2003-07-10 Nautilus Biotech S.A. Mutant recombinant adeno-associated viruses
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
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences 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
NZ535100A (en) 2002-03-20 2008-04-30 Univ Florida 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
AU2003237159A1 (en) 2002-04-30 2003-11-17 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
AU2003265855A1 (en) 2002-08-28 2004-03-19 University Of Florida Modified aav
GB0220467D0 (en) 2002-09-03 2002-10-09 Oxford Biomedica Ltd Composition
US20100144038A1 (en) 2003-03-04 2010-06-10 Masato Miyake Composition And Method For Increasing Efficiency Of Introduction Of Target Substance Into Cell
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
PT2657248T (pt) 2003-06-19 2017-06-26 Genzyme Corp Viriões aav com imunorreatividade diminuída e seus usos
WO2005000240A2 (en) 2003-06-23 2005-01-06 A & G Pharmaceutical, Inc. Compositions and methods for restoring sensitivity of tumor cells to antitumor therapy and inducing apoptosis
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
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
WO2005030932A2 (en) 2003-09-24 2005-04-07 The Board Of Trustees Of The Leland Stanford Junior University Igf-1 instructs multipotent adult cns neural stem cells to an oligodendroglial lineage
EP2292780B1 (en) 2003-09-30 2017-08-23 The Trustees Of The University Of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
WO2005072364A2 (en) 2004-01-27 2005-08-11 University Of Florida A 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
NZ590132A (en) 2004-10-21 2012-03-30 Genentech Inc Dosage regime for treating intraocular neovascular diseases with VEGF
ES2384391T3 (es) * 2004-11-08 2012-07-04 Chromagenics B.V. Selección de células hospedantes que expresan proteína a altos niveles
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
WO2006101634A1 (en) 2005-02-17 2006-09-28 The Regents Of The University Of California Müller cell specific gene therapy
US7922670B2 (en) 2005-02-24 2011-04-12 Warren Jones System and method for quantifying and mapping visual salience
EP2359865B1 (en) 2005-04-07 2013-10-02 The Trustees of The University of Pennsylvania Method of increasing the function of an AAV vector
US20060234347A1 (en) 2005-04-13 2006-10-19 Harding Thomas C Targeting multiple angiogenic pathways for cancer therapy using soluble tyrosine kinase receptors
CN101287837B (zh) 2005-10-20 2015-03-18 尤尼克尔生物制药股份有限公司 昆虫细胞中生产的改进的aav载体
US20090191597A1 (en) 2006-01-20 2009-07-30 Asklepios Biopharmaceutical, Inc. Enhanced production of infectious parvovirus vectors in insect cells
WO2007089632A2 (en) 2006-01-27 2007-08-09 The 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
EP2007795B1 (en) 2006-03-30 2016-11-16 The Board Of Trustees Of The Leland Stanford Junior University 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
EP2012826B1 (en) 2006-05-04 2016-04-13 Fovea Pharmaceuticals Combination comprising a vegf inhibitor and a serine protease for treating neovascular diseases
EP2019588A4 (en) 2006-05-04 2010-11-24 Univ Wayne State RESTORATION OF VISUAL RESPONSES BY IN VIVO ADMINISTRATION OF NUCLEIC ACIDS OF RHODOPSIN
ES2390499T3 (es) 2006-06-12 2012-11-13 Opko Pharmaceuticals, Llc Composiciones y métodos para la inhibición de la angiogénesis por sirna
GB0612096D0 (en) 2006-06-19 2006-07-26 Greater Glasgow Nhs Board Functional imaging of the retina
US8512981B2 (en) 2006-06-21 2013-08-20 Amsterdam Molecular Therapeutics B.V. Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV
JPWO2008001543A1 (ja) 2006-06-27 2009-11-26 株式会社アドバンテスト 半導体試験装置および半導体メモリの試験方法
DK2061891T3 (da) 2006-08-24 2012-07-23 Virovek Inc Ekspression i insektceller af gener med overlappende åbne læserammer, fremgangsmåder og sammensætninger hertil
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
US8075137B2 (en) 2007-02-16 2011-12-13 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
WO2008150459A1 (en) 2007-05-30 2008-12-11 The Trustees Of The University Of Pennsylvania A 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
EP2227553A4 (en) 2007-11-30 2011-01-19 Scarab Genomics Llc LAC-EXPRESSION SYSTEM
CA2715924C (en) 2008-02-19 2021-01-12 Andrew Christian BAKKER Optimisation of expression of parvoviral rep and cap proteins in insect cells
BRPI0908496A2 (pt) 2008-02-20 2019-01-15 Genzyme Corp inibição de angiogênese
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
US20120093772A1 (en) 2008-05-20 2012-04-19 Alan Horsager Vectors for delivery of light sensitive proteins and methods of use
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
US9192685B2 (en) 2008-10-07 2015-11-24 Bracco Suisse S.A. Targeting construct comprising anti-polymer antibody and contrast/therapeutic agents binding to the same
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
BRPI1013364A2 (pt) 2009-03-04 2016-03-29 Deutsches Krebsforsch proteína ativadora de montagem (aap) e seu uso para a fabricação de partículas de parvovírus que consistem essencialmente em vp3.
WO2010114948A2 (en) 2009-04-02 2010-10-07 University Of Florida Research Foundation, Inc. An 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
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel 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
US20120172419A1 (en) 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
EP2524037B1 (en) 2010-01-12 2018-05-16 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
SG183929A1 (en) 2010-03-29 2012-10-30 Univ Pennsylvania Pharmacologically induced transgene ablation system
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
WO2011140450A1 (en) 2010-05-07 2011-11-10 Medicinelodge, Inc Dba Imds Co-Innovation Surgical rasp with radiofrequency ablation
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
AU2011329872B2 (en) 2010-11-16 2017-04-13 Excelimmune, Inc. Methods for producing 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
DK2699270T3 (en) 2011-04-22 2017-10-02 Univ California ADENO-ASSOCIATED VIRUS VIRUSES WITH VARIANT CAPSID AND METHODS FOR USING IT
WO2013029030A1 (en) 2011-08-24 2013-02-28 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
JP6385920B2 (ja) 2012-05-09 2018-09-05 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター
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
JP6363958B2 (ja) 2012-12-25 2018-07-25 タカラバイオ株式会社 Ａａｖ変異体
WO2014186160A1 (en) * 2013-05-16 2014-11-20 Applied Genetic Technologies Corporation Promoters, expression cassettes, vectors, kits, and methods for the threatment of achromatopsia and other diseases
US11685935B2 (en) 2013-05-29 2023-06-27 Cellectis Compact scaffold of Cas9 in the type II CRISPR system
ES2897508T3 (es) 2013-05-31 2022-03-01 Univ California Variantes de virus adenoasociados y métodos de uso de las mismas
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
EP3564379A1 (en) 2013-09-13 2019-11-06 California Institute of Technology Selective recovery
US20160369298A1 (en) 2013-09-26 2016-12-22 University Of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
CN115141258B (zh) 2013-10-11 2025-05-20 马萨诸塞眼科耳科诊所 预测祖先病毒序列的方法及其用途
WO2015058048A1 (en) 2013-10-18 2015-04-23 Regeneron Pharmaceuticals, Inc. Methods and compositions comprising a combination of a vegf antagonist and an anti-ctla-4 antibody
ES2768763T3 (es) 2014-03-04 2020-06-23 Univ Florida Vectores rAAV mejorados y métodos para la transducción de fotorreceptores y células EPR
JP6689249B2 (ja) 2014-03-17 2020-05-20 アドヴェラム バイオテクノロジーズ， インコーポレイテッド 錐体細胞における増強された遺伝子発現のための組成物および方法
PE20170260A1 (es) 2014-05-02 2017-04-12 Genzyme Corp Vectores de aav para la terapia genica de la retina y el snc
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
AU2016226289B2 (en) 2015-03-02 2021-04-29 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
HUE051491T2 (hu) 2015-03-06 2021-03-01 Massachusetts Eye & Ear Infirmary Gén-augmentációs terápiák a PRPF31 gén mutációi által okozott öröklött retina degenerációra
US10883117B2 (en) 2015-03-24 2021-01-05 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US20190000940A1 (en) 2015-07-31 2019-01-03 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
WO2017190125A1 (en) 2016-04-29 2017-11-02 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
WO2017192584A1 (en) 2016-05-03 2017-11-09 Adverum Biotechnologies, Inc. Organotypic pig retina culture system
DK4206216T3 (da) 2016-05-13 2025-12-01 4D Molecular Therapeutics Inc Adeno-associerede virusvarianter og fremgangsmåder til brug deraf
LT3471780T (lt) 2016-06-16 2021-01-25 Adverum Biotechnologies, Inc. Amd gydymas, naudojant aav2 variantą su afliberceptu
EP4427808A3 (en) 2016-06-16 2024-10-30 Adverum Biotechnologies, Inc. Compositions and methods for reducing ocular neovascularization
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
AU2018227483A1 (en) 2017-02-28 2019-09-12 Adverum Biotechnologies, Inc. Modified AAV capsids and uses thereof
CA3054942A1 (en) 2017-03-17 2018-09-20 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
CA3059995A1 (en) 2017-08-28 2019-03-07 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof

Also Published As

Publication number Publication date
CN106414474B (zh) 2021-01-15
US12275959B2 (en) 2025-04-15
AU2020200948B2 (en) 2021-12-09
US11248214B2 (en) 2022-02-15
WO2015142941A1 (en) 2015-09-24
AU2022200708A1 (en) 2022-02-24
KR20160135754A (ko) 2016-11-28
EP3119798B1 (en) 2020-08-05
IL259321B2 (en) 2023-10-01
ES3042252T3 (en) 2025-11-19
EP3800191A1 (en) 2021-04-07
US20180320145A1 (en) 2018-11-08
US10000741B2 (en) 2018-06-19
JP2019205465A (ja) 2019-12-05
US20220259568A1 (en) 2022-08-18
EP3800191B1 (en) 2025-08-20
ES2822551T3 (es) 2021-05-04
CA2942776A1 (en) 2015-09-24
CN112626125A (zh) 2021-04-09
KR102288849B1 (ko) 2021-08-12
AU2020200948A1 (en) 2020-02-27
KR102537394B1 (ko) 2023-05-30
JP2022037158A (ja) 2022-03-08
CN106414474A (zh) 2017-02-15
JP2017510296A (ja) 2017-04-13
AU2015231439B2 (en) 2019-11-14
NZ724403A (en) 2021-02-26
EP3119798A4 (en) 2018-04-04
IL259321A (en) 2018-07-31
IL259321B1 (en) 2023-06-01
IL247756B (en) 2018-05-31
JP2022037159A (ja) 2022-03-08
EP3119798A1 (en) 2017-01-25
KR20210100746A (ko) 2021-08-17
US20150259395A1 (en) 2015-09-17
CA2942776C (en) 2023-01-24
JP6689249B2 (ja) 2020-05-20
SG10201810150UA (en) 2018-12-28
AU2015231439A1 (en) 2016-10-06
EP3800191C0 (en) 2025-08-20

Similar Documents

Publication Publication Date Title
IL259321A (en) Preparations and methods for increasing gene expression in frogs
IL251951A0 (en) Methods and compositions for natural killer cells
IL251576A0 (en) Methods and compositions for creating or maintaining multifunctional cells
ZA201900225B (en) Methods and compositions for gene expression in plants
EP3212225A4 (en) Methods and compositions for modified t cells
GB2535253B (en) Compositions and methods
EP3215168A4 (en) Altering gene expression in modified t cells and uses thereof
EP3151846A4 (en) Methods and compositions for nuclease design
SG11201606934SA (en) Improved reprogramming methods and cell culture platforms
EP3215139A4 (en) Compositions and methods for improved car-t cell therapies
SG11201609916RA (en) Culture method and cell mass
ZA201703443B (en) Oral rehydration composition and methods thereof
GB201417828D0 (en) New methods and compositions
PL3586821T3 (pl) Kompozycje stymulujące komórki macierzyste i sposoby
EP3107996A4 (en) Tscm cells and methods for use
EP3177302A4 (en) Compositions and methods for the reprogramming of cells into cardiomyocytes
IL250178A0 (en) Methods and preparations for the expression of peptides in the cell
SG11201702897WA (en) Cell culture bag and cell culture method
IL257524A (en) Short interfering RNA and its use in methods and preparations for inhibiting the expression of ankyrin horseradish protein gene regulated by Nutz
GB201401373D0 (en) Compositions for cells lysis and uses thereof
EP3258978A4 (en) Methods and products for delivering cells
AU2021203981B2 (en) Altering gene expression in CART cells and uses thereof
GB201402915D0 (en) Compositions and methods
AU2015900510A0 (en) Methods and products for delivering cells
GB201403212D0 (en) Compositions and methods