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

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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
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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，
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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
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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)

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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

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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

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JP2022082310A Withdrawn JP2022107042A (ja) 2015-03-02 2022-05-19 ポリヌクレオチドを網膜錐体に硝子体内送達するための組成物及び方法

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CN (2) CN114480502A (enExample)
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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
MX349138B (es) 2011-04-22 2017-07-13 Univ California Variones de virus adeno-asociados con capside variante y sus metodos de uso.
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
JP7095994B2 (ja) 2015-03-02 2022-07-05 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 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
CA2979065A1 (en) 2015-03-24 2016-09-29 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
GB201508026D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Capsid
CA2996420A1 (en) 2015-09-28 2017-04-06 The University Of North Carolina At Chapel Hill Methods and compositions for antibody-evading virus vectors
GB2545763A (en) 2015-12-23 2017-06-28 Adverum Biotechnologies Inc Mutant viral capsid libraries and related systems and methods
FI3795181T3 (fi) * 2016-06-16 2025-10-14 Adverum Biotechnologies Inc Amd:n hoito aav2-variantilla ja afliberseptillä
CN116286986A (zh) 2016-07-29 2023-06-23 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
EP3528785A4 (en) * 2016-10-19 2020-12-02 Adverum Biotechnologies, Inc. MODIFIED AAV CASPIDS AND USES THEREOF
JP2020503997A (ja) * 2017-01-03 2020-02-06 ビトリーン, インク.Vitrean, Inc. 網膜剥離の治療の方法及び装置
EP3570895A1 (en) * 2017-01-17 2019-11-27 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods of expressing a polynucleotide of interest in the cone photoreceptors
EP4653536A2 (en) 2017-03-17 2025-11-26 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression
AU2018290954B2 (en) * 2017-06-30 2023-07-13 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
EP3676373A4 (en) 2017-08-28 2021-06-09 The Regents of The University of California Adeno-associated virus capsid variants and methods of use thereof
KR20250011726A (ko) * 2017-09-20 2025-01-21 4디 몰레큘러 테라퓨틱스 아이엔씨. 아데노-관련 바이러스 변이체 캡시드 및 그 용도
AU2018350700B2 (en) * 2017-10-16 2025-04-24 Vigeneron Gmbh AAV vectors
WO2019077159A1 (en) 2017-10-20 2019-04-25 INSERM (Institut National de la Santé et de la Recherche Médicale) METHODS OF EXPRESSING POLYNUCLEOTIDE OF INTEREST IN CONE PHOTORECCEPTORS OF A SUBJECT COMPRISING SUB-RETINAL ADMINISTRATION OF A THERAPEUTICALLY EFFECTIVE AMOUNT OF A RECOMBINANT AAV9 DERIVATIVE VECTOR
GB201800546D0 (en) 2018-01-12 2018-02-28 Ucl Business Plc Treatment
SG11202009451VA (en) 2018-04-03 2020-10-29 Stridebio Inc Antibody-evading virus vectors
CN112272672A (zh) 2018-04-03 2021-01-26 斯特里迪比奥公司 抗体逃避性病毒载体
CA3094465A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
US20210290433A1 (en) * 2018-07-20 2021-09-23 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Intraocular delivery 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
US20220096658A1 (en) * 2018-12-21 2022-03-31 The United States Of America, As Represented By The Secretary, Department Of Health And Human Svcs. Adeno-associated viruses and their uses for inner ear therapy
US12270033B2 (en) 2019-02-15 2025-04-08 Exhaura, Ltd. Dual leucine zipper kinase inhibitors for gene therapy
JP7597380B2 (ja) * 2019-03-04 2024-12-10 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 対側眼へのａａｖ遺伝子治療の逐次的硝子体内投与
SG11202110146VA (en) 2019-03-21 2021-10-28 Stridebio Inc Recombinant adeno-associated virus vectors
TWI862583B (zh) * 2019-04-26 2024-11-21 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
EP4045525A1 (en) 2019-10-17 2022-08-24 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
CA3157464A1 (en) * 2019-10-31 2021-05-06 Universitat Bern Aav vector variants for ocular gene delivery
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
TW202227632A (zh) 2020-08-19 2022-07-16 美商史崔德生物公司 用於治療雷特症候群之腺相關病毒載體
US12385064B2 (en) * 2021-03-26 2025-08-12 Adverum Biotechnologies, Inc. Intravitreal dosing for delivery of polynucleotides to retinal cones
US20240252679A1 (en) * 2021-05-28 2024-08-01 Shanghai Regenelead Therapies Co., Ltd. Recombinant adeno-associated virus having variant capsid, and application thereof
WO2022271984A1 (en) * 2021-06-24 2022-12-29 Applied Genetic Technologies Corporation Methods for the treatment of achromatopsia and other cngb3 associated diseases
MX2024004217A (es) * 2021-10-08 2024-06-26 Dyno Therapeutics Inc Variantes de cápside y métodos de uso de estas.
JP2025501286A (ja) * 2021-12-28 2025-01-17 成都弘基生物科技有限公司 修飾されたａａｖカプシドタンパク質及びその使用
CA3255976A1 (en) * 2022-04-15 2023-10-19 Dyno Therapeutics Inc CAPSIDE VARIANTS AND THEIR METHODS OF USE
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
JP2755817B2 (ja) 1988-11-14 1998-05-25 アメリカ合衆国 パルボウイルスキャプシド
FR2643252B1 (fr) 1989-02-21 1991-06-07 Technomed Int Sa Appareil de destruction selective de cellules incluant les tissus mous et les os a l'interieur du corps d'un etre vivant par implosion de bulles de gaz
US5112946A (en) 1989-07-06 1992-05-12 Repligen Corporation Modified pf4 compositions and methods of use
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US6287815B1 (en) 1989-09-14 2001-09-11 Rijksuniversiteit Te Leiden Human parvovirus B19 proteins and virus-like particles, their production and their use in diagnostic assays and vaccines
CA2096222C (en) 1990-11-13 1998-12-29 Stephen D. Lupton Bifunctional selectable fusion genes
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
AU684498B2 (en) 1993-03-25 1997-12-18 Merck & Co., Inc. Inhibitor of vascular endothelial cell growth factor
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
CA2251738A1 (en) 1996-04-16 1997-10-23 Immusol Incorporated Targeted viral vectors
EP1003530A4 (en) 1996-08-20 2001-10-04 Univ California EYE TREATMENT USING SYNTHETIC THYROID HORMONE COMPOSITIONS
WO1998013071A1 (en) 1996-09-24 1998-04-02 Merck & Co., Inc. Gene therapy for inhibition of angiogenesis
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
ES2224375T3 (es) 1997-04-14 2005-03-01 Cell Genesys, Inc. Metodos para aumentar la eficacia del producto de aav recombinante.
WO1998051323A1 (en) 1997-05-13 1998-11-19 The Regents Of The University Of California Novel antiangiogenic peptide agents and their therapeutic and diagnostic use
US6710036B2 (en) 1997-07-25 2004-03-23 Avigen, Inc. Induction of immune response to antigens expressed by recombinant adeno-associated virus
US6458157B1 (en) 1997-08-04 2002-10-01 Suaning Gregg Joergen Retinal stimulator
WO1999014354A1 (en) 1997-09-19 1999-03-25 The Trustees Of The University Of The Pennsylvania Methods and vector constructs useful for production of recombinant aav
JPH11100327A (ja) 1997-09-26 1999-04-13 Toray Ind Inc 網膜変性症治療剤
EP1705248A1 (en) 1997-10-01 2006-09-27 G.D. Searle LLC. Fusion proteins comprising an angiostatin moiety and their use in anti-tumour treatment
AU1449499A (en) 1997-10-31 1999-05-24 Maxygen, Incorporated Modification of virus tropism and host range by viral genome shuffling
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
US6410300B1 (en) 1998-01-12 2002-06-25 The University Of North Carolina At Chapel Hill Methods and formulations for mediating adeno-associated virus (AAV) attachment and infection and methods for purifying AAV
EP1045921A2 (en) 1998-01-16 2000-10-25 Chiron Corporation Feline immunodeficiency virus gene therapy vectors
US6551795B1 (en) 1998-02-18 2003-04-22 Genome Therapeutics Corporation Nucleic acid and amino acid sequences relating to pseudomonas aeruginosa for diagnostics and therapeutics
ES2186340T3 (es) 1998-03-12 2003-05-01 Genentech Inc Utilizacion de polipeptidos fgf-5 para la prevencion de la muerte de neuronas retinales y para el tratamiento de enfermedades oculares.
DE19827457C1 (de) 1998-06-19 2000-03-02 Medigene Ag Strukturprotein von AAV, seine Herstellung und Verwendung
US6593133B1 (en) 1998-07-06 2003-07-15 Nsgene A/S Neurotrophic factors
AU764686B2 (en) 1998-08-28 2003-08-28 Duke University Adenoviruses deleted in the IVa2, 100K, polymerase and/or preterminal protein sequences
JP2002525064A (ja) 1998-09-11 2002-08-13 ザ レジェンツ オブ ザ ユニヴァースティ オブ カリフォルニア 心臓で組織特異的に発現する新規組換えアデノウイルス
EP1114171A1 (en) 1998-09-17 2001-07-11 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
CA2348382C (en) 1998-11-10 2013-09-17 The University Of North Carolina At Chapel Hill Chimeric parvovirus 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
EP1183051A2 (en) 1999-03-15 2002-03-06 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
US7087411B2 (en) 1999-06-08 2006-08-08 Regeneron Pharmaceuticals, Inc. Fusion protein capable of binding VEGF
US7070959B1 (en) 1999-06-08 2006-07-04 Regeneron Pharmaceuticals, Inc. Modified chimeric polypeptides with improved pharmacokinetic properties
US7306799B2 (en) 1999-06-08 2007-12-11 Regeneron Pharmaceuticals, Inc. Use of VEGF inhibitors for treatment of eye disorders
US7314912B1 (en) 1999-06-21 2008-01-01 Medigene Aktiengesellschaft AAv scleroprotein, production and use thereof
DE19933288A1 (de) 1999-07-15 2001-01-18 Medigene Ag Strukturprotein von Adeno-assoziiertem Virus mit veränderter Antigenität, seine Herstellung und Verwendung
DE19933719A1 (de) 1999-07-19 2001-01-25 Medigene Ag Strukturprotein in Adeno-assoziiertem Virus mit veränderten chromatographischen Eigenschaften, seine Herstellung und Verwendung
US8232081B2 (en) 1999-09-21 2012-07-31 Basf Se Methods and microorganisms for production of panto-compounds
WO2001034018A2 (en) 1999-10-25 2001-05-17 Therus Corporation Use of focused ultrasound for vascular sealing
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
WO2001078586A1 (en) 2000-04-17 2001-10-25 Stuart Graham Method and apparatus for objective electrophysiological assessment of visual function
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
AU2002236988A1 (en) 2001-02-06 2002-08-19 Novartis Ag Photodynamic therapy of occult choroidal neovascularization linked to age-related macular degeneration
EP1372552B1 (de) 2001-03-27 2017-03-01 WaveLight GmbH Vorrichtung zur bearbeitung und diagnose von augengewebe
EP1386004A4 (en) 2001-04-05 2005-02-16 Ribozyme Pharm Inc MODULATION OF GENE EXPRESSION ASSOCIATED WITH INFLAMMATORY PROLIFERATION AND GROWTH OF NEURITIES BY METHODS INVOLVING NUCLEIC ACID
WO2002082904A2 (en) 2001-04-13 2002-10-24 The Trustees Of The University Of Pennsylvania Method of treating or retarding the development of blindness
JP2002363107A (ja) 2001-06-04 2002-12-18 Noriyuki Azuma 色覚不全動物の色覚復元方法
DK1412493T3 (da) 2001-08-02 2012-01-09 Inst Clayton De La Rech Fremgangsmåder og sammensætninger der angår forbedrede lentiviral-vektor.-produktionsystemer
US7647184B2 (en) 2001-08-27 2010-01-12 Hanall Pharmaceuticals, Co. Ltd High throughput directed evolution by rational mutagenesis
US20030129203A1 (en) 2001-08-27 2003-07-10 Nautilus Biotech S.A. Mutant recombinant adeno-associated viruses
US6723551B2 (en) 2001-11-09 2004-04-20 The United States Of America As Represented By The Department Of Health And Human Services Production of adeno-associated virus in insect cells
NZ618298A (en) 2001-11-13 2015-04-24 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
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
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
WO2003093436A2 (en) 2002-04-30 2003-11-13 University Of Florida Treatment for phenylketonuria
AU2003223775A1 (en) 2002-04-30 2003-11-17 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
WO2004020600A2 (en) 2002-08-28 2004-03-11 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
WO2004083441A2 (en) 2003-03-19 2004-09-30 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Random peptide library displayed on aav vectors
WO2004108922A2 (en) 2003-04-25 2004-12-16 The Trustees Of The University Of Pennsylvania Use of aav comprising a capsid protein from aav7 or aav8 for the delivery of genes encoding apoprotein a or e genes to the liver
EP1486567A1 (en) 2003-06-11 2004-12-15 Deutsches Krebsforschungszentrum Stiftung des öffentlichen Rechts Improved adeno-associated virus (AAV) vector for gene therapy
EP2657248B1 (en) 2003-06-19 2017-03-22 Genzyme Corporation AAV virions with decreased immunoreactivity and uses therefor
US7368428B2 (en) 2003-06-23 2008-05-06 A&G Pharmaceutical, Inc. Compositions and methods for restoring sensitivity of tumor cells to antitumor therapy and inducing apoptosis
US9233131B2 (en) 2003-06-30 2016-01-12 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
US9441244B2 (en) 2003-06-30 2016-09-13 The Regents Of The University Of California Mutant adeno-associated virus virions and methods of use thereof
AU2004276316A1 (en) 2003-09-24 2005-04-07 The Board Of Trustees Of The Leland Stanford Junior University IGF-1 instructs multipotent adult CNS neural stem cells to an oligodendroglial lineage
HUE035567T2 (en) 2003-09-30 2018-05-28 Univ Pennsylvania Adeno-associated virus (AAV) clusters, sequences, vectors and applications containing them
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
EP2319492A3 (en) 2004-10-21 2011-08-10 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
NZ555830A (en) 2004-12-15 2009-01-31 Univ North Carolina Chimeric vectors
US20100172871A1 (en) 2005-02-17 2010-07-08 Flannery John G Muller Cell Specific Gene Therapy
US7922670B2 (en) 2005-02-24 2011-04-12 Warren Jones System and method for quantifying and mapping visual salience
EP1866422B1 (en) 2005-04-07 2016-04-06 The Trustees of The University of Pennsylvania Method of increasing the function of an aav vector
US20060234347A1 (en) 2005-04-13 2006-10-19 Harding Thomas C Targeting multiple angiogenic pathways for cancer therapy using soluble tyrosine kinase receptors
PT3272872T (pt) 2005-10-20 2020-06-26 Uniqure Ip Bv Vetores aav melhorados produzidos em células de insetos
WO2007084773A2 (en) 2006-01-20 2007-07-26 University Of North Carolina At Chapel Hill Enhanced production of infectious parvovirus vectors in insect cells
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
US20070190028A1 (en) 2006-02-13 2007-08-16 Jihong Qu Method and apparatus for heat or electromagnetic control of gene expression
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
EP2019588A4 (en) 2006-05-04 2010-11-24 Univ Wayne State RESTORATION OF VISUAL RESPONSES BY IN VIVO ADMINISTRATION OF NUCLEIC ACIDS OF RHODOPSIN
CN101437543A (zh) 2006-05-04 2009-05-20 佛维雅制药股份有限公司 用于治疗新生血管病的包含vegf抑制剂和丝氨酸蛋白酶的组合
DK2029746T3 (da) 2006-06-12 2012-10-08 Exegenics Inc D B A Opko Health Inc Sammensætninger og fremgangsmåder til siRNA-hæmning af angiogenese
GB0612096D0 (en) 2006-06-19 2006-07-26 Greater Glasgow Nhs Board Functional imaging of the retina
AU2007261806B2 (en) 2006-06-21 2013-08-15 Uniqure Ip B.V. Vectors with modified initiation codon for the translation of AAV-Rep78 useful for production of AAV in insect cells
JPWO2008001543A1 (ja) 2006-06-27 2009-11-26 株式会社アドバンテスト 半導体試験装置および半導体メモリの試験方法
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
KR20100016462A (ko) 2007-04-13 2010-02-12 카탈리스트 바이오사이언시즈, 인코포레이티드 변형 제ｖｉｉ 인자 폴리펩타이드 및 이의 용도
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
US20110053274A1 (en) 2007-11-30 2011-03-03 Scarab Genomics Llc Lac expression system
AU2009215987B2 (en) 2008-02-19 2015-01-22 Uniqure Ip B.V. Optimisation of expression of parvoviral rep and cap proteins in insect cells
BRPI0908496A2 (pt) 2008-02-20 2019-01-15 Genzyme Corp inibição de angiogênese
US20100081707A1 (en) 2008-02-21 2010-04-01 Ali Robin R 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
WO2010011404A2 (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
CA2748995C (en) 2008-10-07 2018-01-16 Bracco Suisse Sa Targeting construct comprising anti-polymer antibody and liposomes or microvesicles binding to the same
ES2634118T3 (es) 2009-02-11 2017-09-26 The University Of North Carolina At Chapel Hill Vectores de virus modificados y métodos para fabricar y utilizar los mismos
CN102341406B (zh) 2009-03-04 2016-06-08 德国癌症研究中心 组装活化蛋白（aap）及其用于制备基本上由vp3组成的细小病毒颗粒的应用
US8679837B2 (en) 2009-04-02 2014-03-25 University Of Florida Research Foundation, Inc. Inducible system for highly efficient production of recombinant Adeno-associated virus (rAAV) vectors
CN101532024A (zh) 2009-04-30 2009-09-16 许瑞安 一种用于基因治疗的新型细胞特异性内含microRNA结合序列的基因HAAVmir
US8734809B2 (en) 2009-05-28 2014-05-27 University Of Massachusetts AAV's and uses thereof
TWI466158B (zh) 2009-07-03 2014-12-21 Univ Lunghwa Sci & Technology 電漿測量裝置、電漿系統及測量電漿特性之方法
EP2287323A1 (en) 2009-07-31 2011-02-23 Association Institut de Myologie Widespread gene delivery to the retina using systemic administration of AAV vectors
EP2292781A1 (en) 2009-08-17 2011-03-09 Genethon Baculovirus-based production of biopharmaceuticals free of contaminating baculoviral virions
US20120172419A1 (en) 2009-09-15 2012-07-05 Medical College Of Wisconsin Research Foundation Inc. Reagents and methods for modulating cone photoreceptor activity
WO2011088081A1 (en) 2010-01-12 2011-07-21 The University Of North Carolina At Chapel Hill Restrictive inverted terminal repeats for viral vectors
EP2545165B1 (en) 2010-03-11 2020-07-29 uniQure 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
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
WO2011140450A1 (en) 2010-05-07 2011-11-10 Medicinelodge, Inc Dba Imds Co-Innovation Surgical rasp with radiofrequency ablation
US8663624B2 (en) 2010-10-06 2014-03-04 The Regents Of The University Of California Adeno-associated virus virions with variant capsid and methods of use thereof
US20110052678A1 (en) 2010-11-05 2011-03-03 Shantha Totada R Method for treating age related macular degeneration
AU2011329872B2 (en) 2010-11-16 2017-04-13 Excelimmune, Inc. Methods for producing recombinant proteins
DK2661494T3 (da) 2011-01-07 2019-09-02 Applied Genetic Tech Corporation Promotorer, ekspressionskassetter og vektorer til anvendelse ved behandling af achromatopsi og andre sygdomme
EP3147295B2 (en) 2011-08-24 2023-11-22 The Board of Trustees of the Leland Stanford Junior University New avv 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
EP3839050A3 (en) 2012-04-18 2021-09-29 The Board of Trustees of the Leland Stanford Junior University Non-disruptive gene targeting
WO2013170078A1 (en) 2012-05-09 2013-11-14 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)
WO2013188316A1 (en) 2012-06-11 2013-12-19 Avalanche Biotechnologies, Inc. Optical regulation of gene expression in the retina
CN104937100B (zh) 2012-12-25 2020-04-03 宝生物工程株式会社 Aav 变体
US11685935B2 (en) 2013-05-29 2023-06-27 Cellectis Compact scaffold of Cas9 in the type II CRISPR system
CA2907799A1 (en) 2013-05-31 2014-12-04 The Regents Of The University Of California Adeno-associated virus variants and methods of use thereof
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
EP3044318B1 (en) 2013-09-13 2019-05-01 California Institute of Technology Selective recovery
EP3049527A4 (en) 2013-09-26 2017-08-16 University of Florida Research Foundation, Inc. Synthetic combinatorial aav capsid library for targeted gene therapy
EP3459965B1 (en) 2013-10-11 2020-12-02 Massachusetts Eye & Ear Infirmary Methods of predicting ancestral virus sequences and uses thereof
CA2926853C (en) 2013-10-18 2022-04-26 Regeneron Pharmaceuticals, Inc. Methods and compositions comprising a combination of a vegf antagonist and an anti-ctla-4 antibody
EP3113787B1 (en) * 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
KR102288849B1 (ko) 2014-03-17 2021-08-12 애드베룸 바이오테크놀로지스, 인코포레이티드 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법
PT3137497T (pt) 2014-05-02 2021-07-12 Genzyme Corp Vetores de aav para terapia genética na retina e snc
CA2951707A1 (en) 2014-06-10 2015-12-17 Massachusetts Institute Of Technology Method for gene editing
JP7095994B2 (ja) 2015-03-02 2022-07-05 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 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
RS60668B1 (sr) 2015-03-06 2020-09-30 Massachusetts Eye & Ear Infirmary Terapije augmentacije gena za naslednu degeneraciju retine uzrokovanu mutacijama u genu prpf31
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
EP3448437B1 (en) 2016-04-29 2021-12-29 Adverum Biotechnologies, Inc. Evasion of neutralizing antibodies by a recombinant adeno-associated virus
RU2725286C2 (ru) 2016-05-13 2020-06-30 4Д Молекьюлар Терапьютикс Инк. Варианты капсидов аденоассоциированного вируса и способы их применения
HRP20220612T1 (hr) 2016-06-16 2022-07-22 Adverum Biotechnologies, Inc. Sastavi i metode za smanjenje očne neovaskularizacije
FI3795181T3 (fi) 2016-06-16 2025-10-14 Adverum Biotechnologies Inc Amd:n hoito aav2-variantilla ja afliberseptillä
EP3528785A4 (en) 2016-10-19 2020-12-02 Adverum Biotechnologies, Inc. MODIFIED AAV CASPIDS AND USES THEREOF
CA3054687A1 (en) 2017-02-28 2018-09-07 Adverum Biotechnologies, Inc. Modified aav capsids and uses thereof
EP4653536A2 (en) 2017-03-17 2025-11-26 Adverum Biotechnologies, Inc. Compositions and methods for enhanced gene expression

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

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

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