AU2015231439B2 - Compositions and methods for enhanced gene expression in cone cells - Google Patents

Compositions and methods for enhanced gene expression in cone cells Download PDF

Info

Publication number
AU2015231439B2
AU2015231439B2 AU2015231439A AU2015231439A AU2015231439B2 AU 2015231439 B2 AU2015231439 B2 AU 2015231439B2 AU 2015231439 A AU2015231439 A AU 2015231439A AU 2015231439 A AU2015231439 A AU 2015231439A AU 2015231439 B2 AU2015231439 B2 AU 2015231439B2
Authority
AU
Australia
Prior art keywords
seq
sequence
cone
polynucleotide
expression
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
AU2015231439A
Other languages
English (en)
Other versions
AU2015231439A1 (en
Inventor
Thomas W. Chalberg
Jay Neitz
Maureen Neitz
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
University of Washington
Adverum Biotechnologies Inc
Original Assignee
University of Washington
Adverum Biotechnologies Inc
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 University of Washington, Adverum Biotechnologies Inc filed Critical University of Washington
Publication of AU2015231439A1 publication Critical patent/AU2015231439A1/en
Application granted granted Critical
Publication of AU2015231439B2 publication Critical patent/AU2015231439B2/en
Priority to AU2020200948A priority Critical patent/AU2020200948B2/en
Priority to AU2022200708A priority patent/AU2022200708A1/en
Active legal-status Critical Current
Anticipated expiration legal-status Critical

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)
AU2015231439A 2014-03-17 2015-03-17 Compositions and methods for enhanced gene expression in cone cells Active AU2015231439B2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
AU2020200948A AU2020200948B2 (en) 2014-03-17 2020-02-10 Compositions and methods for enhanced gene expression in cone cells
AU2022200708A AU2022200708A1 (en) 2014-03-17 2022-02-03 Compositions and methods for enhanced gene expression in cone cells

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

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2020200948A Division AU2020200948B2 (en) 2014-03-17 2020-02-10 Compositions and methods for enhanced gene expression in cone cells

Publications (2)

Publication Number Publication Date
AU2015231439A1 AU2015231439A1 (en) 2016-10-06
AU2015231439B2 true AU2015231439B2 (en) 2019-11-14

Family

ID=54068212

Family Applications (3)

Application Number Title Priority Date Filing Date
AU2015231439A Active AU2015231439B2 (en) 2014-03-17 2015-03-17 Compositions and methods for enhanced gene expression in cone cells
AU2020200948A Active AU2020200948B2 (en) 2014-03-17 2020-02-10 Compositions and methods for enhanced gene expression in cone cells
AU2022200708A Abandoned AU2022200708A1 (en) 2014-03-17 2022-02-03 Compositions and methods for enhanced gene expression in cone cells

Family Applications After (2)

Application Number Title Priority Date Filing Date
AU2020200948A Active AU2020200948B2 (en) 2014-03-17 2020-02-10 Compositions and methods for enhanced gene expression in cone cells
AU2022200708A Abandoned AU2022200708A1 (en) 2014-03-17 2022-02-03 Compositions and methods for enhanced gene expression in cone cells

Country Status (11)

Country Link
US (3) US10000741B2 (enExample)
EP (2) EP3119798B1 (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
US20120172419A1 (en) 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
HRP20220036T1 (hr) 2011-04-22 2022-04-01 The Regents Of The University Of California Virioni adeno-povezanog virusa s varijantom kapsida i postupci njihove upotrebe
TWI698240B (zh) 2012-05-15 2020-07-11 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)
CA2907799A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
KR102288849B1 (ko) 2014-03-17 2021-08-12 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
CN114480502A (zh) * 2015-03-02 2022-05-13 阿德夫拉姆生物技术股份有限公司 用于将多核苷酸玻璃体内递送到视网膜视锥的组合物和方法
JP7042741B2 (ja) 2015-12-03 2022-03-28 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 ＳｙｎＰ１６１、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
EP3384033B1 (en) * 2015-12-03 2021-09-08 Friedrich Miescher Institute for Biomedical Research Synp160, a promoter for the specific expression of genes in rod photoreceptors
AU2016370487C1 (en) 2015-12-14 2022-03-03 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
AU2017257169B2 (en) * 2016-04-29 2021-07-08 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
SMT202200251T1 (it) * 2016-06-16 2022-07-21 Adverum Biotechnologies Inc Composizioni e metodi per ridurre la neovascolarizzazione oculare
BR112019000171A2 (pt) * 2016-07-08 2019-10-01 Univ Pennsylvania métodos e composições para tratamento de distúrbios e doenças envolvendo rdh12
CN114672516B (zh) * 2016-07-29 2024-11-26 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和其使用方法
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
JP7766393B2 (ja) * 2017-06-14 2025-11-10 ザ・トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア 眼疾患のための遺伝子療法
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
AU2018324477A1 (en) 2017-08-28 2019-10-17 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof
PT3684423T (pt) * 2017-09-20 2023-06-09 4D Molecular Therapeutics Inc Cápsides variantes de vírus adeno-associado e métodos de utilização das mesmas
JP7420710B2 (ja) * 2017-10-20 2024-01-23 アンセルム（アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル） 組換えａａｖ９由来ベクターの治療有効量の網膜下送達を含む、対象の錐体視細胞において目的のポリヌクレオチドを発現させる方法
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
EP3758736B1 (en) 2018-03-02 2024-05-01 The United States of America, as represented by The Secretary, Department of Health and Human Services Il-34 for use in a method of treating 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
EP3893717A4 (en) * 2018-12-12 2022-09-14 Tesseract Health, Inc. BIOMETRIC IDENTIFICATION TECHNIQUES
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
CN114340683A (zh) * 2019-04-15 2022-04-12 衣阿华大学研究基金会 用于转基因表达的方法和组合物
JP2022529782A (ja) * 2019-04-19 2022-06-24 ユニバーシティ オブ マサチューセッツ シュタルガルト病（ａｂｃａ４）の遺伝子治療
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
EP4090381A1 (en) 2020-01-17 2022-11-23 The United States of America, as represented by the Secretary, Department of Health and Human Services <smallcaps/>? ? ?crx-? ? ? ? ?gene therapy for treatment ofautosomal dominant retinopathies
AU2021236233A1 (en) * 2020-03-11 2022-09-22 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
US20230398235A1 (en) * 2020-07-23 2023-12-14 President And Fellows Of Harvard College Variant txnip compositions and methods of use thereof for the treatment of degenerative ocular diseases
EP4188450A2 (en) * 2020-07-29 2023-06-07 University Of Florida Research Foundation, Incorporated Improved aav-mediated x-linked retinoschisis therapies
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
IL308832A (en) 2021-05-26 2024-01-01 Sparingvision G protein mediates increased opening and closing of light-sensitive potassium ion channels in rod and 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
CN115997012B (zh) * 2021-09-06 2024-03-08 辉大(上海)生物科技有限公司 Rpe65相关眼睛疾病和障碍的治疗
US20230338581A1 (en) 2021-10-11 2023-10-26 Sparingvision G-protein-gated-k+ channel-mediated enhancements in light sensitivity in 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
EP4357359A1 (en) 2022-10-20 2024-04-24 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

Citations (1)

* 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

Family Cites Families (193)

* 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
AU631159B2 (en) 1988-11-14 1992-11-19 United States of America, as represented by the Secretary, U.S. Department of Commerce, The 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
US6379885B1 (en) 1989-09-14 2002-04-30 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
JP3734262B2 (ja) 1993-03-25 2006-01-11 メルク エンド カンパニー インコーポレーテッド 血管内皮細胞増殖因子阻害剤
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
JPH10501681A (ja) 1994-02-22 1998-02-17 ダナ−ファーバー キャンサー インスティチュート 核酸送達システムならびにその合成および使用方法
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
JP3345428B2 (ja) 1996-08-20 2002-11-18 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 合成甲状腺ホルマン組成物を使用した眼の処置
ATE343400T1 (de) 1996-09-24 2006-11-15 Merck & Co Inc Verbindungen zur hemmung der angiogenese durch gentherapie
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
JP4135120B2 (ja) 1997-04-14 2008-08-20 セル ジェネシス，インコーポレーテッド 組換えａａｖ生産の効率を上昇させる方法
EP1173191A4 (en) 1997-05-13 2004-12-01 Univ California NEW ANTIANGIOGENIC PEPTIDAGENTS 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
CA2318575A1 (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
ATE226444T1 (de) 1998-03-12 2002-11-15 Genentech Inc Verwendung von fgf-5 polypetiden zur vorbeugung des absterbens retinaler neuronen und behandlung okularer krankheiten
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
EP1108049A2 (en) 1998-08-28 2001-06-20 Duke University Adenoviruses deleted in the iva2, 100k and/or preterminal protein sequences
HK1039795A1 (zh) 1998-09-11 2002-05-10 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
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
EP1135468B1 (en) 1998-11-10 2010-01-06 University Of North Carolina At Chapel Hill 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
AU3755900A (en) 1999-03-15 2000-10-04 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
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
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
AU2619301A (en) 1999-10-25 2001-06-06 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
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
EP1357912B9 (en) 2001-02-06 2012-06-13 QLT Inc. Photodynamic therapy of occult choroidal neovascularization linked to age-related macular degeneration
US20040102765A1 (en) 2001-03-27 2004-05-27 Karsten Koenig Method for the minimal-to non-invase optical treatment of tissues of the eye and for diagnosis thereof and device for carrying out said method
EP1381276A4 (en) 2001-04-13 2005-02-02 Univ Pennsylvania METHOD FOR TREATMENT OR DEVELOPMENT SLUDGE DEGRADATION
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
KR101014207B1 (ko) 2001-11-13 2011-02-14 더 트러스티스 오브 더 유니버시티 오브 펜실바니아 아데노-결합 바이러스 (ａａｖ) 서열을 검출 및/또는 확인하는 방법 및 그 방법에 의해 확인된 신규한 서열을 분리하는 방법
EP1456383B1 (en) 2001-12-21 2014-03-12 Medigene AG A library of modified structural genes or capsid modified particles useful for the identification of viral clones with desired cell tropism
US20060193830A1 (en) 2002-03-20 2006-08-31 Hauswirth William W Raav vector compositions and methods for the treatment of choroidal neovascularization
AU2003221733A1 (en) 2002-04-17 2003-11-03 University Of Florida Research Foundation, Inc. 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
US20070172460A1 (en) 2003-03-19 2007-07-26 Jurgen Kleinschmidt 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
PT2657247T (pt) 2003-06-19 2017-06-29 Genzyme Corp Viriões aav com imunorreatividade diminuída e seus usos
JP2007524622A (ja) 2003-06-23 2007-08-30 エイアンドジー ファーマスーティカルズ インコーポレイテッド 抗癌治療に対する腫瘍細胞の感受性を回復させてアポトーシスを誘導するための組成物及び方法
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
US20050148069A1 (en) 2003-09-24 2005-07-07 Gage Fred H. IGF-1 instructs multipotent adult CNS neural stem cells to an oligodendroglial lineage
EP2298926A1 (en) 2003-09-30 2011-03-23 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
WO2006047325A1 (en) 2004-10-21 2006-05-04 Genentech, Inc. Method for treating intraocular neovascular diseases
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
CN101203613B (zh) 2005-04-07 2012-12-12 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
JP5305913B2 (ja) 2005-10-20 2013-10-02 アムステルダム モレキュラー セラピューティクス ビー．ブイ． 昆虫細胞で産生される改良されたａａｖベクター
WO2007084773A2 (en) 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill 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
US7384145B2 (en) 2006-02-16 2008-06-10 The Board Of Trustees Of The University Of Illinois Mapping retinal function using corneal electrode array
US8118752B2 (en) 2006-02-16 2012-02-21 The Board Of Trustees Of The University Of Illinois Apparatus and methods for mapping retinal function
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
EP2019588A4 (en) 2006-05-04 2010-11-24 Univ Wayne State RESTORATION OF VISUAL RESPONSES BY IN VIVO ADMINISTRATION OF NUCLEIC ACIDS OF RHODOPSIN
CA2651194A1 (en) 2006-05-04 2007-11-15 Fondation Ophthalmologique Aldolphe De Rothschild Methods for treating neovascular diseases
JP2009540011A (ja) 2006-06-12 2009-11-19 エクセジェニックス、インク．ディー／ビー／エー オプコ ヘルス、インク． 血管新生のｓｉＲＮＡ阻害のための組成物及び方法
GB0612096D0 (en) 2006-06-19 2006-07-26 Greater Glasgow Nhs Board Functional imaging of the retina
CA2655957C (en) 2006-06-21 2016-05-03 Amsterdam Molecular Therapeutics (Amt) B.V. Vectors with modified initiation codon for the translation of aav-rep78 useful for production of aav in insect cells
WO2008001543A1 (en) 2006-06-27 2008-01-03 Advantest Corporation Semiconductor testing apparatus and semiconductor memory testing method
US8945918B2 (en) 2006-08-24 2015-02-03 Virovek, Inc. Expression in insect cells of genes with overlapping open reading frames, methods and compositions therefor
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
PT2147096E (pt) 2007-04-13 2015-07-21 Catalyst Biosciences Inc Polipéptidos do factor vii modificados e suas utilizações
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
WO2009023805A1 (en) 2007-08-16 2009-02-19 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
WO2009073551A2 (en) 2007-11-30 2009-06-11 Scarab Genomics Llc Lac expression system
ES2644880T3 (es) 2008-02-19 2017-11-30 Uniqure Ip B.V. Optimización de expresión de proteínas Rep y Cap parvovirales en las células de insecto
CN101951925A (zh) 2008-02-20 2011-01-19 建新公司 血管发生抑制
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
WO2009137006A2 (en) 2008-04-30 2009-11-12 The University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
AU2009274482A1 (en) 2008-05-20 2010-01-28 Eos Neuroscience, Inc. 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.
US20110171262A1 (en) 2008-06-17 2011-07-14 Andrew Christian Bakker Parvoviral capsid with incorporated gly-ala repeat region
WO2010040772A2 (en) 2008-10-07 2010-04-15 Bracco Research Sa Targeting construct comprising anti-polymer antibody and liposomes or microvesicles binding to the same
US8889641B2 (en) 2009-02-11 2014-11-18 The University Of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
US9464119B2 (en) 2009-03-04 2016-10-11 Deutsches Krebsforschungszentrum Assembly activating protein (AAP) and its use for the manufacture of parvovirus particles essentially consisting of 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
US20110270256A1 (en) 2009-09-24 2011-11-03 Medicinelodge, Inc. Dba Imds Co-Innovation Surgical rasp with radiofrequency ablation
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
EP3792348A3 (en) 2010-03-11 2021-06-23 uniQure IP B.V. Mutated rep encoding sequences for use in aav production
EP2550021A2 (en) 2010-03-22 2013-01-30 Association Institut de Myologie Methods of increasing efficiency of vector penetration of target tissue
WO2011126808A2 (en) 2010-03-29 2011-10-13 The Trustees Of The University Of 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
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
JP6092782B2 (ja) 2010-11-16 2017-03-08 エクセリミューン， インコーポレイテッド 組換えタンパク質の製造方法
EP2661494B1 (en) * 2011-01-07 2019-06-12 Applied Genetic Technologies Corporation Promoters, expression cassettes and vectors for use treating achromatopsia and other diseases
HRP20220036T1 (hr) 2011-04-22 2022-04-01 The Regents Of The University Of California Virioni adeno-povezanog virusa s varijantom kapsida i postupci njihove upotrebe
ES2857773T5 (es) 2011-08-24 2024-06-04 Univ Leland Stanford Junior Nuevas proteínas de la cápside de AAV para la transferencia de ácidos nucleicos
WO2013063601A1 (en) 2011-10-28 2013-05-02 University Of Florida Research Foundation, Inc. Chimeric promoter for cone photoreceptor targeted gene therapy
EP3470523A1 (en) 2012-05-09 2019-04-17 Oregon Health & Science University Adeno associated virus plasmids and vectors
TWI698240B (zh) 2012-05-15 2020-07-11 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
EP2940131B1 (en) 2012-12-25 2019-02-20 Takara Bio Inc. Aav variant
JP6588008B2 (ja) * 2013-05-16 2019-10-09 アプライド ジェネティック テクノロジーズ コーポレイション １色覚および他の疾患の治療のためのプロモーター、発現カセット、ベクター、キット、ならびに方法
JP6670743B2 (ja) 2013-05-29 2020-03-25 セレクティスＣｅｌｌｅｃｔｉｓ Ｉｉ型ｃｒｉｓｐｒシステムにおける新規のコンパクトなｃａｓ９足場
CA2907799A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
US9597415B2 (en) 2013-06-28 2017-03-21 Inserm (Institute National De La Sante Et De La Recherche Medicale) Methods for expressing a polynucleotide of interest in the retina of a subject
US20150025939A1 (en) 2013-07-18 2015-01-22 Somnath Chatterjee Company Centric Social Media Platfonn for Content Sharing aud Tracking
EP3561062A1 (en) 2013-09-13 2019-10-30 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
CN115141260B (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
JP6607850B2 (ja) 2013-10-18 2019-11-20 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Ｖｅｇｆアンタゴニスト及び抗ｃｔｌａ−４抗体の組み合わせを含む方法及び組成物
CA2941640A1 (en) 2014-03-04 2015-09-11 University Of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
KR102288849B1 (ko) 2014-03-17 2021-08-12 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
JP6741591B2 (ja) 2014-05-02 2020-08-19 ジェンザイム・コーポレーション 網膜およびｃｎｓ遺伝子治療用ａａｖベクター
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
CN114480502A (zh) 2015-03-02 2022-05-13 阿德夫拉姆生物技术股份有限公司 用于将多核苷酸玻璃体内递送到视网膜视锥的组合物和方法
US20180043034A1 (en) 2015-03-06 2018-02-15 Massachusetts Eye And Ear Infirmary Gene augmentation therapies for inherited retinal degeneration caused by mutations in the prpf31 gene
JP6836999B2 (ja) 2015-03-24 2021-03-03 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニアＴｈｅ Ｒｅｇｅｎｔｓ Ｏｆ Ｔｈｅ Ｕｎｉｖｅｒｓｉｔｙ Ｏｆ Ｃａｌｉｆｏｒｎｉａ アデノ随伴ウイルス変異体及びその使用方法
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
AU2017257169B2 (en) 2016-04-29 2021-07-08 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
EP3452065A4 (en) 2016-05-03 2019-11-20 Adverum Biotechnologies, Inc. ORGANIC PIG TRAINING SYSTEM
CN116333057A (zh) 2016-05-13 2023-06-27 4D分子治疗有限公司 腺相关病毒变体衣壳和其使用方法
HRP20201842T1 (hr) 2016-06-16 2021-04-16 Adverum Biotechnologies, Inc. Liječenje amd korištenjem aav2 varijante s afliberceptom
SMT202200251T1 (it) 2016-06-16 2022-07-21 Adverum Biotechnologies Inc Composizioni e metodi per ridurre la neovascolarizzazione oculare
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
CA3054687A1 (en) 2017-02-28 2018-09-07 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
AU2018324477A1 (en) 2017-08-28 2019-10-17 The Regents Of The University Of California Adeno-associated virus capsid variants and methods of use thereof

Patent Citations (1)

* 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

Also Published As

Publication number Publication date
WO2015142941A1 (en) 2015-09-24
JP2022037159A (ja) 2022-03-08
US10000741B2 (en) 2018-06-19
EP3800191C0 (en) 2025-08-20
EP3119798A1 (en) 2017-01-25
AU2022200708A1 (en) 2022-02-24
IL259321A (en) 2018-07-31
KR102288849B1 (ko) 2021-08-12
CA2942776C (en) 2023-01-24
AU2015231439A1 (en) 2016-10-06
CN106414474A (zh) 2017-02-15
JP2019205465A (ja) 2019-12-05
US12275959B2 (en) 2025-04-15
IL247756B (en) 2018-05-31
EP3119798A4 (en) 2018-04-04
AU2020200948B2 (en) 2021-12-09
EP3800191A1 (en) 2021-04-07
AU2020200948A1 (en) 2020-02-27
NZ724403A (en) 2021-02-26
US20180320145A1 (en) 2018-11-08
JP6689249B2 (ja) 2020-05-20
JP2022037158A (ja) 2022-03-08
JP2017510296A (ja) 2017-04-13
EP3119798B1 (en) 2020-08-05
CN106414474B (zh) 2021-01-15
US20220259568A1 (en) 2022-08-18
EP3800191B1 (en) 2025-08-20
SG10201810150UA (en) 2018-12-28
CN112626125A (zh) 2021-04-09
KR102537394B1 (ko) 2023-05-30
IL259321B2 (en) 2023-10-01
US11248214B2 (en) 2022-02-15
CA2942776A1 (en) 2015-09-24
ES2822551T3 (es) 2021-05-04
KR20160135754A (ko) 2016-11-28
IL259321B1 (en) 2023-06-01
ES3042252T3 (en) 2025-11-19
US20150259395A1 (en) 2015-09-17
SG11201607738WA (en) 2016-10-28
KR20210100746A (ko) 2021-08-17

Similar Documents

Publication Publication Date Title
US12275959B2 (en) Compositions and methods for enhanced gene expression in cone cells
US20210388030A1 (en) Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
US20200399656A1 (en) Compositions and methods for enhancing functional expression of therapeutic genes in photoreceptors
JP2025503495A (ja) 錐体光受容体細胞を産生するためのプロセス
HK40049243A (en) Compositions and methods for enhanced gene expression in cone cells
HK1229816A1 (en) Compositions and methods for enhanced gene expression in cone cells
HK1229816B (en) Compositions and methods for enhanced gene expression in cone cells
NZ724403B2 (en) Compositions and methods for enhanced gene expression in cone cells
JP2025500979A (ja) 光受容体細胞のインビボリプログラミング

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)