JP7095994B2 - ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法 - Google Patents

ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法 Download PDF

Info

Publication number
JP7095994B2
JP7095994B2 JP2017546102A JP2017546102A JP7095994B2 JP 7095994 B2 JP7095994 B2 JP 7095994B2 JP 2017546102 A JP2017546102 A JP 2017546102A JP 2017546102 A JP2017546102 A JP 2017546102A JP 7095994 B2 JP7095994 B2 JP 7095994B2
Authority
JP
Japan
Prior art keywords
pyramidal
expression
retinal
cells
amino acids
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
JP2017546102A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018508519A (ja
JP2018508519A5 (OSRAM
Inventor
トーマス ダブリュー． チャルベルク，
ジェイ ナイツ，
モーリーン ナイツ，
Original Assignee
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
ユニヴァーシティ オブ ワシントン
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 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, ユニヴァーシティ オブ ワシントン filed Critical 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
Publication of JP2018508519A publication Critical patent/JP2018508519A/ja
Publication of JP2018508519A5 publication Critical patent/JP2018508519A5/ja
Priority to JP2022082310A priority Critical patent/JP2022107042A/ja
Application granted granted Critical
Publication of JP7095994B2 publication Critical patent/JP7095994B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

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
    • 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
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the delivery route, e.g. oral, subcutaneous
    • 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
    • A61P27/02—Ophthalmic agents
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/861—Adenoviral vectors
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Virology (AREA)
  • Biophysics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Ophthalmology & Optometry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2017546102A 2015-03-02 2016-03-02 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法 Active JP7095994B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022082310A JP2022107042A (ja) 2015-03-02 2022-05-19 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201562127194P 2015-03-02 2015-03-02
US62/127,194 2015-03-02
US201562134466P 2015-03-17 2015-03-17
US62/134,466 2015-03-17
PCT/US2016/020482 WO2016141078A1 (en) 2015-03-02 2016-03-02 Compositions and methods for intravitreal delivery of polynucleotides to retinal cones

Related Child Applications (2)

Application Number Title Priority Date Filing Date
JP2020081106A Division JP2020122008A (ja) 2015-03-02 2020-05-01 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法
JP2022082310A Division JP2022107042A (ja) 2015-03-02 2022-05-19 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法

Publications (3)

Publication Number Publication Date
JP2018508519A JP2018508519A (ja) 2018-03-29
JP2018508519A5 JP2018508519A5 (OSRAM) 2019-04-11
JP7095994B2 true JP7095994B2 (ja) 2022-07-05

Family

ID=56848956

Family Applications (3)

Application Number Title Priority Date Filing Date
JP2017546102A Active JP7095994B2 (ja) 2015-03-02 2016-03-02 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法
JP2020081106A Pending JP2020122008A (ja) 2015-03-02 2020-05-01 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法
JP2022082310A Withdrawn JP2022107042A (ja) 2015-03-02 2022-05-19 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法

Family Applications After (2)

Application Number Title Priority Date Filing Date
JP2020081106A Pending JP2020122008A (ja) 2015-03-02 2020-05-01 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法
JP2022082310A Withdrawn JP2022107042A (ja) 2015-03-02 2022-05-19 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法

Country Status (15)

Country Link
US (2) US11021519B2 (OSRAM)
EP (1) EP3265178A4 (OSRAM)
JP (3) JP7095994B2 (OSRAM)
KR (1) KR20170137730A (OSRAM)
CN (2) CN107405507B (OSRAM)
AU (2) AU2016226289B2 (OSRAM)
BR (1) BR112017018846A2 (OSRAM)
CA (1) CA2978068C (OSRAM)
EA (1) EA201791939A1 (OSRAM)
HK (1) HK1249067A1 (OSRAM)
IL (1) IL254148B (OSRAM)
MX (1) MX2017011041A (OSRAM)
MY (1) MY187898A (OSRAM)
SG (1) SG11201707063TA (OSRAM)
WO (1) WO2016141078A1 (OSRAM)

Families Citing this family (47)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
SMT202400520T1 (it) 2011-04-22 2025-01-14 Univ California Virioni di virus adeno-associato con capside variante e relativi metodi di utilizzo
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)
CN105247044B (zh) 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
CN106414474B (zh) 2014-03-17 2021-01-15 阿德夫拉姆生物技术股份有限公司 用于视锥细胞中增强的基因表达的组合物和方法
CA2978068C (en) 2015-03-02 2024-05-28 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
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
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
ES2865487T3 (es) 2015-09-28 2021-10-15 Univ North Carolina Chapel Hill Métodos y composiciones para vectores virales que evaden los anticuerpos
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
MX391779B (es) * 2016-06-16 2025-03-21 Adverum Biotechnologies Inc Variante de aav2 con aflibercept para usarse en el tratamiento de una condición o enfermedad ocular.
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
CN110167494A (zh) * 2017-01-03 2019-08-23 维特安公司 用于治疗视网膜脱离的方法和装置
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
MX2019011040A (es) 2017-03-17 2020-01-20 Adverum Biotechnologies Inc Composiciones y metodos para potenciar la expresion genica.
BR112019019015A2 (pt) 2017-06-30 2020-04-14 Univ California vírions de vírus adeno-associado com capsídeos variantes e seus métodos de uso
KR20200039617A (ko) 2017-08-28 2020-04-16 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 아데노-연관 바이러스 캡시드 변이체 및 이의 사용 방법
UA127455C2 (uk) 2017-09-20 2023-08-30 4Д Молекьюлар Терапьютикс Інк. Капсид аденоасоційованого вірусу і спосіб його застосування
CA3078277A1 (en) * 2017-10-16 2019-04-25 Vigeneron Gmbh Aav vectors
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
GB201800546D0 (en) * 2018-01-12 2018-02-28 Ucl Business Plc Treatment
SG11202009452WA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
AU2019247191A1 (en) 2018-04-03 2020-10-15 Ginkgo Bioworks, Inc. Virus vectors for targeting ophthalmic tissues
JP2021519581A (ja) 2018-04-03 2021-08-12 ストライドバイオ，インコーポレイテッド 抗体を回避するウイルスベクター
EP3823567B1 (en) * 2018-07-20 2025-06-18 The USA, as represented by The Secretary, Department of Health and Human Services Intraocular injection of gene therapy expression vectors
CN111118016B (zh) 2018-10-30 2022-04-15 上海朗昇生物科技有限公司 治疗视网膜色素变性疾病的基因治疗载体
WO2020132578A1 (en) * 2018-12-21 2020-06-25 Chien Wade Wei De Adeno-associated viruses and their uses for inner ear therapy
EP3924470A4 (en) 2019-02-15 2022-12-14 Exhaura, Ltd. DUAL LEUCINE ZIPPER KINASE INHIBITORS FOR GENE THERAPY
EP3934698A1 (en) * 2019-03-04 2022-01-12 Adverum Biotechnologies, Inc. Sequential intravitreal administration of aav gene therapy to contralateral eyes
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
TW202532428A (zh) * 2019-04-26 2025-08-16 美商愛德維仁生物科技公司 用於玻璃體內遞送之變異體aav蛋白殼
CA3157700A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
JP7791546B2 (ja) * 2019-10-31 2025-12-24 ウニベルジテート ベルン 眼遺伝子送達のためのａａｖベクター変異体
TWI769795B (zh) 2020-04-27 2022-07-01 美商４Ｄ分子療法公司 密碼子優化的ｇｌａ基因及其用途
CN116249772A (zh) * 2020-05-29 2023-06-09 加利福尼亚大学董事会 具有变体衣壳的腺相关病毒病毒体及其使用方法
CA3189878A1 (en) 2020-08-19 2022-02-24 Colin O'BANION Adeno-associated virus vectors for treatment of rett syndrome
US12385064B2 (en) * 2021-03-26 2025-08-12 Adverum Biotechnologies, Inc. Intravitreal dosing for delivery of polynucleotides to retinal cones
TW202313096A (zh) * 2021-05-28 2023-04-01 大陸商江蘇恆瑞醫藥股份有限公司 衣殼變異的重組腺相關病毒及其應用
WO2022271984A1 (en) * 2021-06-24 2022-12-29 Applied Genetic Technologies Corporation Methods for the treatment of achromatopsia and other cngb3 associated diseases
AU2022358779A1 (en) * 2021-10-08 2024-04-18 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
AU2022423978A1 (en) * 2021-12-28 2024-07-18 Chengdu Origen Biotechnology Co., Ltd. Modified aav capsid protein and use thereof
CN119630688A (zh) * 2022-04-15 2025-03-14 迪诺治疗股份有限公司 衣壳变体及其使用方法
CN116970648A (zh) * 2022-04-24 2023-10-31 上海朗昇生物科技有限公司 新型aav衣壳改造株及其用途
EP4598941A2 (en) * 2022-09-13 2025-08-13 Dyno Therapeutics, Inc. Capsid variants and methods of using the same
WO2024110770A1 (en) * 2022-11-22 2024-05-30 INSERM (Institut National de la Santé et de la Recherche Médicale) A new promoter for retinal pigment epithelium (rpe) targeted gene therapy
CN119350454B (zh) * 2024-12-27 2025-03-25 上海朗昇生物科技有限公司 衣壳重排型腺相关病毒载体及其用途

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014518614A (ja) 2011-04-22 2014-08-07 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 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法
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

Family Cites Families (191)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US46016A (en) * 1865-01-24 Improvement in coats with inner sleeves
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
DE69128037T2 (de) 1990-11-13 1998-05-07 Immunex Corp., Seattle, Wash. 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
CA2158745C (en) 1993-03-25 2007-06-19 Richard L. Kendall Inhibitor of vascular endothelial cell growth factor
CA2163427A1 (en) 1993-05-21 1994-12-08 Stephen D. Lupton 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
WO1995022618A1 (en) 1994-02-22 1995-08-24 Dana-Farber Cancer Institute Nucleic acid delivery system, method of synthesis and uses thereof
US6551618B2 (en) 1994-03-15 2003-04-22 University Of Birmingham Compositions and methods for delivery of agents for neuronal regeneration and survival
FR2718150B1 (fr) 1994-03-29 1996-04-26 Rhone Poulenc Rorer Sa Virus recombinants, préparation et utilisation en thérapie génique.
US5639725A (en) 1994-04-26 1997-06-17 Children's Hospital Medical Center Corp. Angiostatin protein
US5527533A (en) 1994-10-27 1996-06-18 Board Of Trustees Of The University Of Illinois Method of retarding and ameliorating central nervous system and eye damage
US20020168342A1 (en) 1994-11-03 2002-11-14 Cell Genesys, Inc. Novel adenoviral vectors, packaging cell lines, recombinant adenoviruses and methods
US6440425B1 (en) 1995-05-01 2002-08-27 Aventis Pasteur Limited High molecular weight major outer membrane protein of moraxella
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
EP0927044A4 (en) 1996-04-16 1999-09-08 Immusol Inc DEFINED TARGET VIRAL VECTORS
WO1998007435A1 (en) 1996-08-20 1998-02-26 The Regents Of The University Of California Eye treatments using synthetic thyroid hormone compositions
DE69736860T2 (de) 1996-09-24 2007-05-16 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
CA2287478C (en) 1997-04-14 2007-06-19 Richard J. Samulski Methods for increasing the efficiency of recombinant aav product
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
AU9319198A (en) 1997-09-19 1999-04-05 Trustees Of The University Of Pennsylvania, The 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
EP1019515A1 (en) 1997-10-01 2000-07-19 G.D. SEARLE & CO. 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
WO1999036511A2 (en) 1998-01-16 1999-07-22 Chiron Corporation Feline immunodeficiency virus gene therapy vectors
US6551795B1 (en) 1998-02-18 2003-04-22 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to pseudomonas aeruginosa for diagnostics and therapeutics
EP1061943B1 (en) 1998-03-12 2002-10-23 Genentech, Inc. Use of fgf-5 polypeptides for 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
WO2000012740A2 (en) 1998-08-28 2000-03-09 Duke University ADENOVIRUSES DELETED IN THE IVa2, 100K AND/OR PRETERMINAL PROTEIN SEQUENCES
DE69925134T2 (de) 1998-09-11 2006-01-19 The Regents Of The University Of California, La Jolla Rekombinante adenovirus für gewebespezifische genexpression ins herz
CA2346262A1 (en) 1998-09-17 2000-03-23 University Of Florida Methods for treatment of degenerative retinal diseases
US20040234505A1 (en) 1998-09-23 2004-11-25 Stuart Naylor Polynucleotide constructs and uses thereof
US6491907B1 (en) 1998-11-10 2002-12-10 The University Of North Carolina At Chapel Hill Recombinant parvovirus vectors and method of making
AU3755900A (en) 1999-03-15 2000-10-04 Chiron Corporation Use of recombinant gene delivery vectors for treating or preventing diseases of the eye
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
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
JP2003513691A (ja) 1999-10-25 2003-04-15 シーラス、コーポレイション 血管を封止するための集束超音波の使用
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
ATE511784T1 (de) 2000-04-17 2011-06-15 Univ Sydney Verfahren zur objektiven elektrophysiologischen bewertung der visuellen funktion
US6329181B1 (en) 2000-08-07 2001-12-11 Neurologix, Inc. Helper functions for recombinant vector production
US6962815B2 (en) 2001-01-05 2005-11-08 Children's Hopital Inc. AAV2 vectors and methods
US7749492B2 (en) 2001-01-05 2010-07-06 Nationwide Children's Hospital, Inc. AAV vectors and methods
ATE554763T1 (de) 2001-02-06 2012-05-15 Quadra Logic Tech Inc Photodynamische therapie der mit der altersbezogenen makuladegeneration verbundenen okkulten chorioidalen neovaskularisation
EP1372552B1 (de) 2001-03-27 2017-03-01 WaveLight GmbH Vorrichtung zur bearbeitung und diagnose von augengewebe
CA2442670A1 (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
ATE527347T1 (de) 2001-08-02 2011-10-15 Inst Clayton De La Rech Verfahren und zusammensetzungen im zusammenhang mit verbesserten lentivirusvektor- produktionssystemen
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
ATE317916T1 (de) 2001-11-13 2006-03-15 Univ Pennsylvania Verfahren zur identifizierung von adeno- assoziiertem virus (aav) sequenzen sowie kit zur ausführung der methode
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
AU2003237159A1 (en) 2002-04-30 2003-11-17 University Of Florida Treatment for phenylketonuria
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
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
WO2004079332A2 (en) 2003-03-04 2004-09-16 National Institute Of Advanced Industrial Science And Technology Composition and method for increasing efficiency of introduction of target substance into cell
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
DK2357189T3 (en) 2003-06-19 2017-06-19 Genzyme Corp AAV virions with reduced immunoreactivity and applications thereof
EP1644038A2 (en) 2003-06-23 2006-04-12 A & G Pharmaceuticals, Inc. Compositions and methods for restoring sensitivity of tumor cells to antitumor therapy and inducing apoptosis
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
EP1670897A4 (en) 2003-09-24 2008-02-27 Univ Leland Stanford Junior IGF-1 FORM OF NERVOUS STEM CELLS OF THE CENTRAL NERVOUS SYSTEM ADULTS PLURIPOTENT TO AN OLIGODENDROGICAL LINE
ES2874298T3 (es) 2003-09-30 2021-11-04 Univ Pennsylvania Clados de virus adenoasociados (AAV), secuencias, vectores que contienen el mismo, y usos de los mismos
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
US7427396B2 (en) 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
AU2005299701B2 (en) 2004-10-21 2011-11-17 Genentech, Inc. Method for treating intraocular neovascular diseases
US20060172382A1 (en) 2004-11-08 2006-08-03 Chromagenics B.V. Selection of host cells expressing protein at high levels
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
ES2428218T3 (es) 2005-04-07 2013-11-06 The Trustees Of The University Of Pennsylvania Cápsides de AAV rh48 modificadas, composiciones que las contienen y usos de las mismas
US20060234347A1 (en) 2005-04-13 2006-10-19 Harding Thomas C Targeting multiple angiogenic pathways for cancer therapy using soluble tyrosine kinase receptors
EP1945779B1 (en) 2005-10-20 2013-03-06 UniQure IP B.V. Improved aav vectors produced in insect cells
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
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
US7588772B2 (en) 2006-03-30 2009-09-15 Board Of Trustees Of The Leland Stamford Junior University AAV capsid library and AAV capsid proteins
US8216575B2 (en) 2006-03-31 2012-07-10 Chengdu Kanghong Biotechnologies Co., Ltd. Inhibition of neovascularization with a soluble chimeric protein comprising VEGF FLT-1 and KDR domains
JP2009535371A (ja) 2006-05-04 2009-10-01 フォベア、ファルマスティカル、ソシエテ、アノニム 血管新生性疾患の治療のための方法
EP3437473A1 (en) 2006-05-04 2019-02-06 Wayne State University Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
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
CN101506369B (zh) 2006-06-21 2014-02-12 尤尼克尔生物制药股份有限公司 具有经修饰的用于在昆虫细胞中产生aav的aav-rep78翻译起始密码子的载体
KR100922422B1 (ko) 2006-06-27 2009-10-16 가부시키가이샤 어드밴테스트 반도체 시험 장치 및 반도체 메모리의 시험 방법
ES2385679T3 (es) 2006-08-24 2012-07-30 Virovek, Inc. Expresión de células de insecto de genes con marcos de lectura abiertos superpuestos, métodos y composiciones de éstos
CN1966082B (zh) 2006-11-03 2010-06-30 许瑞安 一种防治结直肠癌的基因药物及其制备方法和用途
AU2008215181A1 (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
UA101155C2 (ru) 2007-04-13 2013-03-11 Каталист Байосайенс, Инк. Модифицированный полипептид фактора vii (fvii) и его применение
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
JP2011512156A (ja) 2008-02-19 2011-04-21 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． 昆虫細胞におけるパルボウイルスｒｅｐ及びｃａｐタンパク質の発現の最適化
WO2009105669A2 (en) 2008-02-20 2009-08-27 Genzyme Corporation Angiogenesis inhibition
WO2009105690A2 (en) 2008-02-21 2009-08-27 Targeted Genetics Corporation Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea
ITFI20080081A1 (it) 2008-04-18 2009-10-19 Strumenti Oftalmici C S O S R Procedimento e sistema per la registrazione di risposte elettrofunzionali erg, perg e vep multifocali in tempo reale
US8632764B2 (en) 2008-04-30 2014-01-21 University Of North Carolina At Chapel Hill Directed evolution and in vivo panning of virus vectors
MX2010012592A (es) 2008-05-20 2011-05-05 Eos Neuroscience Inc Vectores para la administracion de proteinas sensibles a la luz y metodos de uso de las mismas.
ES2330826B1 (es) 2008-06-04 2010-07-26 Proyecto De Biomedicina Cima, S.L. Sistema para empaquetamiento de adenovirus de alta capacidad.
WO2009154452A1 (en) 2008-06-17 2009-12-23 Amsterdam Molecular Therapeutics B.V. Parvoviral capsid with incorporated Gly-Ala repeat region
JP5491511B2 (ja) 2008-10-07 2014-05-14 ブラッコ・シュイス・ソシエテ・アノニム 抗ポリマー抗体およびそれと結合するリポソームまたは微小胞を含むターゲッティング構築物
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
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
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
CN101532024A (zh) 2009-04-30 2009-09-16 许瑞安 一种用于基因治疗的新型细胞特异性内含microRNA结合序列的基因HAAVmir
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
US9169494B2 (en) 2010-01-12 2015-10-27 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
WO2011112089A2 (en) 2010-03-11 2011-09-15 Amsterdam Molecular Therapeutics (Amt) Ip B.V. Mutated rep encoding sequences for use in aav production
US9546112B2 (en) 2010-03-22 2017-01-17 Association Institut De Myologie Methods of increasing efficiency of vector penetration of target tissue
US20130023033A1 (en) 2010-03-29 2013-01-24 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
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
WO2012068317A2 (en) 2010-11-16 2012-05-24 Excelimmune, Inc. Methods for producing recombinant proteins
AU2012204266C1 (en) 2011-01-07 2017-12-21 Applied Genetic Technologies Corporation Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
US9169299B2 (en) 2011-08-24 2015-10-27 The Board Of Trustees Of The Leleand Stanford Junior University 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
ES2828663T3 (es) 2012-04-18 2021-05-27 Univ Leland Stanford Junior Dirección genética no disruptiva
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
WO2014103957A1 (ja) 2012-12-25 2014-07-03 タカラバイオ株式会社 Ａａｖ変異体
US11685935B2 (en) 2013-05-29 2023-06-27 Cellectis Compact scaffold of Cas9 in the type II CRISPR system
CN105247044B (zh) 2013-05-31 2021-05-07 加利福尼亚大学董事会 腺相关病毒变体及其使用方法
EP3013946B8 (en) * 2013-06-28 2019-04-10 INSERM (Institut National de la Santé et de la Recherche Médicale) 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
EP3633041A3 (en) 2013-09-26 2020-07-29 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
WO2015054653A2 (en) 2013-10-11 2015-04-16 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
AU2014337135B2 (en) 2013-10-18 2019-09-19 Regeneron Pharmaceuticals, Inc. Methods and compositions comprising a combination of a VEGF antagonist and an anti-CTLA-4 antibody
US10308957B2 (en) * 2014-03-04 2019-06-04 University Of Florida Research Foundation, Inc. rAAV vectors and methods for transduction of photoreceptors and RPE cells
CN106414474B (zh) * 2014-03-17 2021-01-15 阿德夫拉姆生物技术股份有限公司 用于视锥细胞中增强的基因表达的组合物和方法
AU2015252797C1 (en) 2014-05-02 2021-04-22 Genzyme Corporation AAV vectors for retinal and CNS gene therapy
WO2015191693A2 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
CA2978068C (en) 2015-03-02 2024-05-28 Adverum Biotechnologies, Inc. Compositions and methods for intravitreal delivery of polynucleotides to retinal cones
LT3265568T (lt) 2015-03-06 2020-08-25 Massachusetts Eye & Ear Infirmary Genų augmentacijos terapijos, skirtos paveldimai tinklainės degeneracijai, sukeltai prpf31 geno mutacijos
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
CA3022600A1 (en) 2016-04-29 2017-11-02 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
EP4209501A1 (en) 2016-05-13 2023-07-12 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
MX391779B (es) 2016-06-16 2025-03-21 Adverum Biotechnologies Inc Variante de aav2 con aflibercept para usarse en el tratamiento de una condición o enfermedad ocular.
RS63282B1 (sr) 2016-06-16 2022-06-30 Adverum Biotechnologies Inc Kompozicije i postupci za smanjenje očne neovaskularizacije
CA3040179A1 (en) 2016-10-19 2018-04-26 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
CN110650733A (zh) 2017-02-28 2020-01-03 阿德夫拉姆生物技术股份有限公司 经过修饰的aav衣壳和其用途
MX2019011040A (es) 2017-03-17 2020-01-20 Adverum Biotechnologies Inc Composiciones y metodos para potenciar la expresion genica.

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014518614A (ja) 2011-04-22 2014-08-07 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 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法
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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Sci Transl Med，2013年，5(189)，189ra76

Also Published As

Publication number Publication date
US11021519B2 (en) 2021-06-01
CA2978068C (en) 2024-05-28
AU2016226289A1 (en) 2017-09-21
US20210388030A1 (en) 2021-12-16
CA2978068A1 (en) 2016-09-09
MY187898A (en) 2021-10-27
JP2018508519A (ja) 2018-03-29
IL254148A0 (en) 2017-10-31
WO2016141078A1 (en) 2016-09-09
MX2017011041A (es) 2018-03-02
EA201791939A1 (ru) 2018-01-31
AU2021209206A1 (en) 2021-08-19
BR112017018846A2 (pt) 2018-07-31
EP3265178A1 (en) 2018-01-10
AU2016226289B2 (en) 2021-04-29
HK1249067A1 (zh) 2018-10-26
SG11201707063TA (en) 2017-09-28
KR20170137730A (ko) 2017-12-13
CN107405507A (zh) 2017-11-28
JP2022107042A (ja) 2022-07-20
CN107405507B (zh) 2022-05-03
IL254148B (en) 2022-09-01
EP3265178A4 (en) 2018-09-05
CN114480502A (zh) 2022-05-13
US20180066022A1 (en) 2018-03-08
JP2020122008A (ja) 2020-08-13

Similar Documents

Publication Publication Date Title
JP7095994B2 (ja) ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法
US12275959B2 (en) Compositions and methods for enhanced gene expression in cone cells
JP6827320B2 (ja) ＬＣＡ−８及び進行性ＲＰを治療するための組換えＡＡＶ−Ｃｒｕｍｂｓホモログ組成物及び方法
JP2021507723A (ja) 視細胞における治療遺伝子の機能的発現を増強するための組成物および方法
JP2021520232A (ja) 黄斑ジストロフィーを処置するための組成物及び方法
HK40049243A (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
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

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190301

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20190301

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20200205

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20200501

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20201104

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210204

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210802

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20211101

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20220302

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220519

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20220602

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20220623

R150 Certificate of patent or registration of utility model

Ref document number: 7095994

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250