ES2482094T3 - Administración extendida de genes a neuronas motoras mediante inyección periférica de vectores AAV - Google Patents

Administración extendida de genes a neuronas motoras mediante inyección periférica de vectores AAV Download PDF

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Publication number
ES2482094T3
ES2482094T3 ES08836776.8T ES08836776T ES2482094T3 ES 2482094 T3 ES2482094 T3 ES 2482094T3 ES 08836776 T ES08836776 T ES 08836776T ES 2482094 T3 ES2482094 T3 ES 2482094T3
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aav
motor neurons
vector
genes
aav vectors
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ES2482094T5 (es
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Martine Barkats
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Centre National de la Recherche Scientifique CNRS
Genethon
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Centre National de la Recherche Scientifique CNRS
Genethon
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
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    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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Abstract

Un vector AAV que comprende un gen de interés para uso en un método para administrar dicho gen de interés a neuronas motoras o células gliales mediante inyección intraperitoneal (i.p.), intramuscular (i.m.) o intravenosa (i.v.), preferiblemente inyección intravenosa, de dicho vector AAV a un sujeto, donde dicho vector AAV es: - un vector AAV9 de cadena doble auto-complementario, o - un vector AAV pseudotipado que comprende un genoma de AAV de cadena doble auto-complementario procedente de un serotipo de AAV diferente del serotipo AAV9 y una cápsida derivada de una cápsida de AAV9.

Description

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Claims (1)

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ES08836776T 2007-10-05 2008-10-03 Administración extendida de genes a neuronas motoras mediante inyección periférica de vectores AAV Active ES2482094T5 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP07301435 2007-10-05
EP07301435A EP2058401A1 (en) 2007-10-05 2007-10-05 Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
PCT/EP2008/063297 WO2009043936A1 (en) 2007-10-05 2008-10-03 Widespread gene delivery to motor neurons using peripheral injection of aav vectors

Publications (2)

Publication Number Publication Date
ES2482094T3 true ES2482094T3 (es) 2014-08-01
ES2482094T5 ES2482094T5 (es) 2024-06-03

Family

ID=39186802

Family Applications (3)

Application Number Title Priority Date Filing Date
ES12180951T Active ES2727034T3 (es) 2007-10-05 2008-10-03 Administración extendida de genes a neuronas motoras mediante inyección periférica de vectores AAV
ES08836776T Active ES2482094T5 (es) 2007-10-05 2008-10-03 Administración extendida de genes a neuronas motoras mediante inyección periférica de vectores AAV
ES19158152T Active ES2924892T3 (es) 2007-10-05 2008-10-03 Administración extendida de genes a neuronas motoras mediante inyección periférica de vectores AAV

Family Applications Before (1)

Application Number Title Priority Date Filing Date
ES12180951T Active ES2727034T3 (es) 2007-10-05 2008-10-03 Administración extendida de genes a neuronas motoras mediante inyección periférica de vectores AAV

Family Applications After (1)

Application Number Title Priority Date Filing Date
ES19158152T Active ES2924892T3 (es) 2007-10-05 2008-10-03 Administración extendida de genes a neuronas motoras mediante inyección periférica de vectores AAV

Country Status (19)

Country Link
US (3) US9926574B2 (es)
EP (5) EP2058401A1 (es)
JP (2) JP5620819B2 (es)
CN (2) CN101883859A (es)
CA (2) CA2701561C (es)
CY (2) CY1125608T1 (es)
DK (2) DK2212424T3 (es)
ES (3) ES2727034T3 (es)
FI (1) FI2212424T4 (es)
FR (2) FR20C1057I2 (es)
HR (2) HRP20220992T3 (es)
HU (2) HUE059864T2 (es)
LT (2) LT3505635T (es)
LU (1) LUC00285I2 (es)
NO (1) NO2022048I1 (es)
PL (2) PL2212424T3 (es)
PT (3) PT2212424E (es)
SI (2) SI3505635T1 (es)
WO (1) WO2009043936A1 (es)

Families Citing this family (114)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
EP2019143A1 (en) * 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
CA3108119C (en) 2008-01-15 2024-01-16 The Board Of Trustees Of The Leland Stanford Junior University Markers of acute myeloid leukemia stem cells
PT2242512T (pt) 2008-01-15 2016-07-18 Univ Leland Stanford Junior Métodos para manipular a fagocitose mediada por cd47
US9217155B2 (en) 2008-05-28 2015-12-22 University Of Massachusetts Isolation of novel AAV'S and uses thereof
US9415121B2 (en) 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
US11219696B2 (en) 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9
JP5879256B2 (ja) 2009-05-02 2016-03-08 ジェンザイム・コーポレーション 神経変性障害のための遺伝子治療
WO2010138263A2 (en) 2009-05-28 2010-12-02 University Of Massachusetts Novel aav 's and uses thereof
US9272053B2 (en) 2010-04-23 2016-03-01 University Of Massachusetts AAV-based treatment of cholesterol-related disorders
EP3567106A1 (en) * 2010-04-23 2019-11-13 University of Massachusetts Cns targeting aav vectors and methods of use thereof
CA2833905C (en) 2010-04-23 2019-09-10 University Of Massachusetts Multicistronic expression constructs
CN107828820B (zh) * 2010-10-27 2022-06-07 学校法人自治医科大学 用于向神经系统细胞导入基因的腺相关病毒粒子
US9226976B2 (en) 2011-04-21 2016-01-05 University Of Massachusetts RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
US20130039888A1 (en) 2011-06-08 2013-02-14 Nationwide Children's Hospital Inc. Products and methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
SMT202000445T1 (it) * 2012-06-19 2020-09-10 Univ Florida Composizioni e metodi per trattare patologie
JP6417322B2 (ja) 2012-06-21 2018-11-07 アソシアシオン・アンスティテュ・ドゥ・ミオロジーAssociation Institut De Myologie 遺伝子治療ベクターの広範な遺伝子送達
BR112015002168A2 (pt) 2012-08-01 2017-11-07 Nationwide Childrens Hospital liberação intratecal de vírus 9 adeno-associado recombinante
US9539307B2 (en) 2012-09-17 2017-01-10 The Research Institute At Nationwide Children's Hospital Compositions and methods for treating amyotrophic lateral sclerosis
KR20160010526A (ko) * 2013-05-15 2016-01-27 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
RS63942B1 (sr) 2013-08-27 2023-02-28 Res Inst Nationwide Childrens Hospital Proizvodi i postupci za lečenje amiotrofične lateralne skleroze
AU2014346987A1 (en) 2013-11-05 2016-06-23 The Research Institute At Nationwide Children's Hospital Compositions and methods for inhibiting NF-kB and SOD-1 to treat amyotrophic lateral sclerosis
JP6822841B2 (ja) 2014-03-09 2021-01-27 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 オルニチントランスカルバミラーゼ(otc)欠損症の処置において有用な組成物
EP2933335A1 (en) * 2014-04-18 2015-10-21 Genethon A method of treating peripheral neuropathies and motor neuron diseases
WO2015187825A2 (en) 2014-06-03 2015-12-10 University Of Massachusetts Compositions and methods for modulating dysferlin expression
US10590420B2 (en) 2014-07-31 2020-03-17 Association Institut De Myologie Treatment of amyotrophic lateral sclerosis
EP3200830B1 (en) 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
US10370432B2 (en) 2014-10-03 2019-08-06 University Of Massachusetts Heterologous targeting peptide grafted AAVS
RU2020140209A (ru) 2014-10-21 2021-01-25 Юниверсити Оф Массачусетс Варианты рекомбинантных aav и их применения
IL292999A (en) 2014-11-14 2022-07-01 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
EP4344741A3 (en) 2014-11-21 2024-08-28 The University of North Carolina at Chapel Hill Aav vectors targeted to the central nervous system
US10876134B2 (en) 2014-12-16 2020-12-29 Board Of Regents Of The University Of Nebraska Gene therapy for juvenile batten disease
WO2016131009A1 (en) 2015-02-13 2016-08-18 University Of Massachusetts Compositions and methods for transient delivery of nucleases
WO2016164642A1 (en) * 2015-04-08 2016-10-13 The United States Of America, As Represented By The Secretary Of Health And Human Services Viral gene therapy as treatment for cholesterol storage disease or disorder
US11046955B2 (en) 2015-04-24 2021-06-29 University Of Massachusetts Modified AAV constructs and uses thereof
WO2017058926A1 (en) * 2015-10-01 2017-04-06 Goleini Inc. Targeted expression of chloride channels and methods of use thereof
DK3365438T3 (da) * 2015-10-21 2021-07-12 Us Health Codon-optimeret reduceret-størrelse atp7a cdna og anvendelser ved behandling af kobbertransportlidelser
EP3364996B1 (en) 2015-10-22 2021-08-25 University of Massachusetts Prostate-targeting adeno-associated virus serotype vectors
WO2017070525A1 (en) 2015-10-22 2017-04-27 University Of Massachusetts Methods and compositions for treating metabolic imbalance in neurodegenerative disease
EP3368054A4 (en) 2015-10-28 2019-07-03 Voyager Therapeutics, Inc. REGULATORY EXPRESSION USING THE ADENO-ASSOCIATED VIRUS (AAV)
WO2017085317A1 (en) * 2015-11-19 2017-05-26 Universitat Autonoma De Barcelona Secreted splicing variant of mammal klotho as a medicament for cognition and behaviour impairments
WO2017136536A1 (en) 2016-02-02 2017-08-10 University Of Massachusetts Method to enhance the efficiency of systemic aav gene delivery to the central nervous system
WO2017139643A1 (en) 2016-02-12 2017-08-17 University Of Massachusetts Anti-angiogenic mirna therapeutics for inhibiting corneal neovascularization
WO2017176929A1 (en) 2016-04-05 2017-10-12 University Of Massachusetts Compositions and methods for selective inhibition of grainyhead-like protein expression
AU2017250298B2 (en) 2016-04-15 2024-10-03 The Trustees Of The University Of Pennsylvania Gene therapy for treating mucopolysaccharidosis type II
WO2017181105A1 (en) 2016-04-15 2017-10-19 University Of Massachusetts Methods and compositions for treating metabolic imbalance
KR102629418B1 (ko) 2016-06-13 2024-01-25 더 유니버시티 오브 노쓰 캐롤라이나 엣 채플 힐 최적화된 cln1 유전자 및 발현 카세트 및 그의 용도
US11882815B2 (en) 2016-06-15 2024-01-30 University Of Massachusetts Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells
CN110650673B (zh) 2016-08-30 2024-04-09 加利福尼亚大学董事会 用于生物医学靶向和递送的方法以及用于实践该方法的装置和系统
US10457940B2 (en) 2016-09-22 2019-10-29 University Of Massachusetts AAV treatment of Huntington's disease
CA3040483A1 (en) 2016-10-13 2018-04-19 University Of Massachusetts Aav capsid designs
CN108524952A (zh) * 2017-03-06 2018-09-14 复旦大学 腺相关病毒aav9及其在制备治疗戈谢氏病的制剂中的用途
SG11201909870SA (en) 2017-05-05 2019-11-28 Voyager Therapeutics Inc Compositions and methods of treating amyotrophic lateral sclerosis (als)
JP7229989B2 (ja) 2017-07-17 2023-02-28 ボイジャー セラピューティクス インコーポレイテッド 軌道アレイガイドシステム
TWI832036B (zh) 2017-08-03 2024-02-11 美商航海家醫療公司 用於aav之遞送之組合物及方法
JP7449223B2 (ja) 2017-09-22 2024-03-13 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 ムコ多糖症ii型を治療するための遺伝子療法
US11434502B2 (en) 2017-10-16 2022-09-06 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (ALS)
EP4454654A3 (en) 2017-10-16 2025-02-19 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis (als)
CN120505370A (zh) * 2017-11-08 2025-08-19 诺华股份有限公司 制备病毒载体的手段和方法及其用途
AU2019215152A1 (en) 2018-02-01 2020-08-20 Homology Medicines, Inc. Adeno-associated virus compositions for restoring PAH gene function and methods of use thereof
US10610606B2 (en) 2018-02-01 2020-04-07 Homology Medicines, Inc. Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
JP7244547B2 (ja) 2018-02-19 2023-03-22 ホモロジー・メディシンズ・インコーポレイテッド F8遺伝子機能を回復させるためのアデノ随伴ウイルス組成物及びその使用の方法
WO2019195423A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Virus vectors for targeting ophthalmic tissues
JP7406677B2 (ja) 2018-04-03 2023-12-28 ギンコ バイオワークス 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
WO2019195444A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
WO2019200016A1 (en) 2018-04-10 2019-10-17 President And Fellows Of Harvard College Aav vectors encoding clarin-1 or gjb2 and uses thereof
WO2019222329A1 (en) 2018-05-15 2019-11-21 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
EP3794126A1 (en) 2018-05-15 2021-03-24 Voyager Therapeutics, Inc. Compositions and methods for the treatment of parkinson's disease
EP3793615A2 (en) 2018-05-16 2021-03-24 Voyager Therapeutics, Inc. Directed evolution of aav to improve tropism for cns
US12228571B2 (en) 2018-06-08 2025-02-18 Novartis Ag Cell-based assay for measuring drug product potency
US12163129B2 (en) 2018-06-08 2024-12-10 University Of Massachusetts Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
WO2020010035A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Cannula system
CA3103963A1 (en) 2018-07-02 2020-01-09 Voyager Therapeutics, Inc. Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord
JP2021530548A (ja) 2018-07-24 2021-11-11 ボイジャー セラピューティクス インコーポレイテッドVoyager Therapeutics, Inc. 遺伝子治療製剤を生産するための系および方法
CN113383010B (zh) 2018-09-28 2025-09-09 沃雅戈治疗公司 具有经工程化改造的启动子的共济蛋白表达构建体及其使用方法
TW202035689A (zh) 2018-10-04 2020-10-01 美商航海家醫療公司 測量病毒載體粒子的效價及強度之方法
WO2020077165A1 (en) 2018-10-12 2020-04-16 Voyager Therapeutics, Inc. Compositions and methods for delivery of aav
JP7590968B2 (ja) 2018-12-05 2024-11-27 アビオナ・セラピュ―ティクス・インコーポレイテッド 遺伝子送達のための組み換えアデノ随伴ウイルスベクター
MX2021008542A (es) 2019-01-18 2021-11-12 Voyager Therapeutics Inc Métodos y sistemas para la producción de partículas de aav.
CA3127019A1 (en) 2019-02-05 2020-08-13 The Trustees Of Princeton University Recombinant nucleic acids containing alphaherpesvirus promoter sequences
CN109706176A (zh) * 2019-03-12 2019-05-03 青岛大学附属医院 可靶向转染背根神经节卫星胶质细胞腺病毒载体构建方法
AR118465A1 (es) 2019-03-21 2021-10-06 Stridebio Inc Vectores de virus adenoasociados recombinantes
AU2020248116B2 (en) * 2019-03-28 2025-12-04 President And Fellows Of Harvard College Engineered adeno-associated (AAV) vectors for transgene expression
US20210009987A1 (en) * 2019-07-10 2021-01-14 Locana, Inc. Rna-targeting knockdown and replacement compositions and methods for use
WO2021034920A1 (en) * 2019-08-20 2021-02-25 St. Jude Children's Research Hospital, Inc. SCHIZOPHRENIA-RELATED MICRODELETION GENE 2510002D24Rik IS ESSENTIAL FOR SOCIAL MEMORY
WO2021041489A1 (en) * 2019-08-29 2021-03-04 The Board Of Trustees Of The Leland Stanford Junior University Adeno-associated viral vectors for crossing the human blood brain barrier
WO2021046155A1 (en) 2019-09-03 2021-03-11 Voyager Therapeutics, Inc. Vectorized editing of nucleic acids to correct overt mutations
AU2020367532A1 (en) 2019-10-17 2022-05-12 Ginkgo Bioworks, Inc. Adeno-associated viral vectors for treatment of Niemann-Pick disease type C
KR20220083708A (ko) 2019-10-17 2022-06-20 스트라이드바이오 인코포레이티드 Aav 전달 카세트
TW202140791A (zh) 2020-01-13 2021-11-01 美商霍蒙拉奇醫藥公司 治療苯酮尿症之方法
BR112022020753A2 (pt) 2020-04-15 2022-12-20 Voyager Therapeutics Inc Compostos de ligação a tau
AU2021273447B2 (en) 2020-05-13 2026-03-05 Voyager Therapeutics, Inc. Redirection of tropism of AAV capsids
TW202208632A (zh) 2020-05-27 2022-03-01 美商同源醫藥公司 用於恢復pah基因功能的腺相關病毒組成物及其使用方法
MX2023002016A (es) 2020-08-19 2023-06-26 Sarepta Therapeutics Inc Vectores de virus adenoasociados para el tratamiento del sindrome de rett.
US12129287B2 (en) 2020-09-14 2024-10-29 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
CN112121179A (zh) * 2020-09-15 2020-12-25 山东兴瑞生物科技有限公司 一种组合物及其在治疗脊髓性肌萎缩症中的应用
US20240141378A1 (en) 2021-03-03 2024-05-02 Voyager Therapeutics, Inc. Controlled expression of viral proteins
CN115708866A (zh) * 2021-08-23 2023-02-24 中国科学院深圳先进技术研究院 骨源因子rankl在制备调控中枢神经系统稳态的药物或药物组合物中的应用
EP4405396A2 (en) 2021-09-20 2024-07-31 Voyager Therapeutics, Inc. Compositions and methods for the treatment of her2 positive cancer
IL311861A (en) 2021-11-02 2024-06-01 Voyager Therapeutics Inc Aav capsid variants and uses thereof
US20250011372A1 (en) 2021-11-17 2025-01-09 Voyager Thrapeutics, Inc. Aav capsid variants and uses thereof
AU2023217698A1 (en) 2022-02-08 2024-07-25 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
EP4532515A1 (en) 2022-06-02 2025-04-09 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
US20250215453A1 (en) 2022-06-28 2025-07-03 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2024011112A1 (en) 2022-07-06 2024-01-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
JP2025528068A (ja) 2022-08-03 2025-08-26 ボイジャー セラピューティクス インコーポレイテッド 血液脳関門を通過させるための組成物及び方法
CN116064671A (zh) * 2022-08-10 2023-05-05 深圳先进技术研究院 一种基因递送系统将基因递送到小胶质细胞的应用
CN120019071A (zh) 2022-09-15 2025-05-16 沃雅戈治疗公司 Tau结合化合物
AU2023417630A1 (en) 2022-12-29 2025-05-15 Voyager Therapeutics, Inc. Compositions and methods for regulating mapt
WO2024226790A1 (en) 2023-04-26 2024-10-31 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
EP4705488A1 (en) 2023-05-04 2026-03-11 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025038430A1 (en) 2023-08-16 2025-02-20 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof
WO2025147436A1 (en) 2024-01-03 2025-07-10 Voyager Therapeutics, Inc. Aav capsid variants and uses thereof

Family Cites Families (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996040954A1 (en) 1995-06-07 1996-12-19 Yale University Oral delivery of adeno-associated viral vectors
US20040076613A1 (en) 2000-11-03 2004-04-22 Nicholas Mazarakis Vector system
FR2756297B1 (fr) 1996-11-22 1999-01-08 Centre Nat Rech Scient Procede de production de virus recombinants
EP1002078A1 (en) 1997-07-31 2000-05-24 Chiron Corporation Method enabling readministration of aav vector via immunosuppresion of host
ATE454445T1 (de) 1998-11-10 2010-01-15 Univ North Carolina Virusvektoren und verfahren für ihre herstellung und verabreichung.
US6498244B1 (en) 1999-05-28 2002-12-24 Cell Genesys, Inc. Adeno-associated virus capsid immunologic determinants
US6399385B1 (en) 1999-09-29 2002-06-04 The Trustees Of The University Of Pennsylvania Methods for rapid PEG-modification of viral vectors, compositions for enhanced gene transduction, compositions with enhanced physical stability, and uses therefor
WO2001036603A2 (en) 1999-11-17 2001-05-25 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
CA2410901C (en) 2000-06-01 2012-03-20 University Of North Carolina At Chapel Hill Methods and compounds for controlled release of recombinant parvovirus vectors
ATE318923T1 (de) * 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
WO2003004660A1 (en) 2001-07-03 2003-01-16 Institut National De La Sante Et De La Recherche Medicale (Inserm) Methods of administering vectors to synaptically connected neurons
SG10202108118RA (en) * 2001-11-13 2021-08-30 Univ Pennsylvania A method of detecting and/or identifying adeno-associated virus (aav) sequences and isolating novel sequences identified thereby
ES2526341T3 (es) 2001-12-17 2015-01-09 The Trustees Of The University Of Pennsylvania Secuencias de serotipo 9 de virus adeno-asociado (AAV), vectores que las contienen, y usos de las mismas
EP1463530A4 (en) 2001-12-19 2006-09-06 Lijun Wang ADENO-ASSOCIATED VIRUS-RELATED DELIVERY OF GDNF TO SKELETAL MUSCLES
AU2002364221B2 (en) * 2001-12-21 2008-07-10 The Salk Institute For Biological Studies Targeted retrograde gene delivery to motor neurons
US6998118B2 (en) * 2001-12-21 2006-02-14 The Salk Institute For Biological Studies Targeted retrograde gene delivery for neuronal protection
US20060018882A1 (en) * 2004-06-21 2006-01-26 Kaemmerer William F Medical devices and methods for delivering compositions to cells
JP2006521825A (ja) * 2003-03-31 2006-09-28 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン rAAV導入を高めるための化合物および方法
JP2006526394A (ja) * 2003-06-03 2006-11-24 ベニテック オーストラリア リミテッド 二本鎖核酸
PL2357189T3 (pl) 2003-06-19 2017-08-31 Genzyme Corporation Wiriony AAV o zmniejszonej immunoreaktywności i ich zastosowanie
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
HUE054805T2 (hu) * 2003-09-30 2021-09-28 Univ Pennsylvania Adeno-asszociált vírus (AAV) kládok, szekvenciák, az azokat tartalmazó vektorok és alkalmazásaik
US7723288B2 (en) * 2003-10-14 2010-05-25 Neurologix, Inc. Methods and compositions for the treatment of neurological disease
US8734810B2 (en) 2003-10-29 2014-05-27 Allergan, Inc. Botulinum toxin treatments of neurological and neuropsychiatric disorders
WO2005056807A2 (en) 2003-12-04 2005-06-23 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services, National Institutes Of Health Bovine adeno-associated viral (baav) vector and uses thereof
US20060166363A1 (en) 2004-01-27 2006-07-27 Sergei Zolotukhin Modified baculovirus expression system for production of pseudotyped rAAV vector
JP2007531537A (ja) * 2004-04-06 2007-11-08 セダーズ−シナイ メディカル センター アポリポタンパク質a−iおよびアポリポタンパク質a−imilanoをコードする組換えアデノ随伴ウイルスベクターによる血管疾患の予防および処置
US20060110364A1 (en) * 2004-08-20 2006-05-25 Ludwig Institute For Cancer Research Vector-mediated delivery of polynucleotides encoding soluble VEGF receptors
US8283151B2 (en) 2005-04-29 2012-10-09 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Isolation, cloning and characterization of new adeno-associated virus (AAV) serotypes
AU2006242129A1 (en) 2005-05-02 2006-11-09 Genzyme Corporation Gene therapy for spinal cord disorders
US7867484B2 (en) 2006-01-27 2011-01-11 University Of North Carolina At Chapel Hill Heparin and heparan sulfate binding chimeric vectors
US20070280906A1 (en) 2006-06-03 2007-12-06 Ognjen Petras Method to generate mirrored adenoassociated viral vectors
CA2680063A1 (en) * 2007-02-23 2008-08-28 University Of Florida Research Foundation, Inc. Compositions and methods for treating glycogen storage diseases
US10696983B2 (en) 2007-05-30 2020-06-30 The Trustees Of The University Of Pennsylvania Method for transducing cells with primary cilia
EP2019143A1 (en) * 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
BRPI0817937A2 (pt) 2007-10-01 2015-04-07 Alcon Res Ltd Entrega mediada por aav autocomplementar de moléculas de rna de interferência para tratar ou prevenir distúrbios oculares
EP2058401A1 (en) * 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
US11219696B2 (en) * 2008-12-19 2022-01-11 Nationwide Children's Hospital Delivery of polynucleotides using recombinant AAV9

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