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Adeno-associated virus vector as a platform for gene therapy delivery

Nature reviews. Drug discovery, 2019-05, Vol.18 (5), p.358-378

2019

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Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice

Nature communications, 2021-04, Vol.12 (1), p.2121-13, Article 2121

2021

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Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo

Nature communications, 2021-11, Vol.12 (1), p.6267-6267, Article 6267

2021

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Worldwide Epidemiology of Neutralizing Antibodies to Adeno-Associated Viruses

The Journal of infectious diseases, 2009-02, Vol.199 (3), p.381-390

2009

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Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties

Nature communications, 2020-06, Vol.11 (1), p.3279-3279, Article 3279

2020

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Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis

Nature communications, 2019-07, Vol.10 (1), p.2958-13, Article 2958

2019

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Adeno-Associated Virus Delivery of Anti-HIV Monoclonal Antibodies Can Drive Long-Term Virologic Suppression

Immunity (Cambridge, Mass.), 2019-03, Vol.50 (3), p.567-575.e5

2019

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Delivery of Adeno-Associated Virus Vectors in Adult Mammalian Inner-Ear Cell Subtypes Without Auditory Dysfunction

Human gene therapy, 2018-04, Vol.29 (4), p.492-506

2018

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Streamlined ex vivo and in vivo genome editing in mouse embryos using recombinant adeno-associated viruses

Nature communications, 2018-01, Vol.9 (1), p.412-12, Article 412

2018

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Down-regulation of microRNA-203-3p initiates type 2 pathology during schistosome infection via elevation of interleukin-33

PLoS pathogens, 2018-03, Vol.14 (3), p.e1006957-e1006957

2018

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Querying Recombination Junctions of Replication-Competent Adeno-Associated Viruses in Gene Therapy Vector Preparations with Single Molecule, Real-Time Sequencing

Viruses, 2023-05, Vol.15 (6), p.1228

2023

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Serotype-dependent recombinant adeno-associated vector (AAV) infection of Epstein-Barr virus-positive B-cells, towards recombinant AAV-based therapy of focal EBV + lymphoproliferative disorders

Virology journal, 2021-11, Vol.18 (1), p.223-223, Article 223

2021

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CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector

Molecular therapy. Nucleic acids, 2016, Vol.5 (7), p.e338-e338, Article e338

2016

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Novel Roles of GATA4/6 in the Postnatal Heart Identified through Temporally Controlled, Cardiomyocyte-Specific Gene Inactivation by Adeno-Associated Virus Delivery of Cre Recombinase

PloS one, 2015-05, Vol.10 (5), p.e0128105-e0128105

2015

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Oncolytic Virus Alphavirus M1: A New and Promising Weapon to Fight Cancer

Human gene therapy, 2021-02, Vol.32 (3-4), p.136-137

2021

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Membranous Bubbles: High-Purity and High-Titer Exosomes as the Potential Solution for Adeno-Associated Viruses to Evade Neutralization?

Human gene therapy, 2021-12, Vol.32 (23-24), p.1427-1429

2021

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Recombinant AAV Vectors for Enhanced Expression of Authentic IgG

PloS one, 2016-06, Vol.11 (6), p.e0158009-e0158009

2016

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Adeno-Associated Viruses Undergo Substantial Evolution in Primates during Natural Infections

Proceedings of the National Academy of Sciences - PNAS, 2003-05, Vol.100 (10), p.6081-6086

2003

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Recombinant Adeno-Associated Virus Serotype 6 (rAAV6) Potently and Preferentially Transduces Rat Astrocytes In vitro and In vivo

Frontiers in cellular neuroscience, 2016-11, Vol.10, p.262-262

2016

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Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis

Human gene therapy, 2018-01, Vol.29 (1), p.42-50

2018

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Titration of Lentivirus Vectors

Cold Spring Harbor protocols, 2018-04, Vol.2018 (4), p.pdb.prot095695

2018

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Production of Recombinant Adeno-Associated Viruses (rAAVs) by Transient Transfection

Cold Spring Harbor protocols, 2020-02, Vol.2020 (2), p.pdb.prot095596

2020

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Development and application of an antigen capture ELISA assay for diagnosis of Japanese encephalitis virus in swine, human and mosquito

Virology journal, 2012-01, Vol.9 (1), p.4-4, Article 4

2012

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Enrichment of Fully Packaged Virions in Column-Purified Recombinant Adeno-Associated Virus (rAAV) Preparations by Iodixanol Gradient Centrifugation Followed by Anion-Exchange Column Chromatography

Cold Spring Harbor protocols, 2020-02, Vol.2020 (2), p.pdb.prot095638

2020

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AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice

Nature (London), 2022-04, Vol.604 (7905), p.343-348

2022

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Adeno-associated virus vector as a platform for gene therapy delivery

Improved prime editors enable pathogenic allele correction and cancer modelling in adult mice

Self-inactivating, all-in-one AAV vectors for precision Cas9 genome editing via homology-directed repair in vivo

Worldwide Epidemiology of Neutralizing Antibodies to Adeno-Associated Viruses

Structural characterization of a novel human adeno-associated virus capsid with neurotropic properties

Bone-targeting AAV-mediated silencing of Schnurri-3 prevents bone loss in osteoporosis

Adeno-Associated Virus Delivery of Anti-HIV Monoclonal Antibodies Can Drive Long-Term Virologic Suppression

Delivery of Adeno-Associated Virus Vectors in Adult Mammalian Inner-Ear Cell Subtypes Without Auditory Dysfunction

Streamlined ex vivo and in vivo genome editing in mouse embryos using recombinant adeno-associated viruses

Down-regulation of microRNA-203-3p initiates type 2 pathology during schistosome infection via elevation of interleukin-33

Querying Recombination Junctions of Replication-Competent Adeno-Associated Viruses in Gene Therapy Vector Preparations with Single Molecule, Real-Time Sequencing

Serotype-dependent recombinant adeno-associated vector (AAV) infection of Epstein-Barr virus-positive B-cells, towards recombinant AAV-based therapy of focal EBV + lymphoproliferative disorders

CNS-restricted Transduction and CRISPR/Cas9-mediated Gene Deletion with an Engineered AAV Vector

Novel Roles of GATA4/6 in the Postnatal Heart Identified through Temporally Controlled, Cardiomyocyte-Specific Gene Inactivation by Adeno-Associated Virus Delivery of Cre Recombinase

Oncolytic Virus Alphavirus M1: A New and Promising Weapon to Fight Cancer

Membranous Bubbles: High-Purity and High-Titer Exosomes as the Potential Solution for Adeno-Associated Viruses to Evade Neutralization?

Recombinant AAV Vectors for Enhanced Expression of Authentic IgG

Adeno-Associated Viruses Undergo Substantial Evolution in Primates during Natural Infections

Recombinant Adeno-Associated Virus Serotype 6 (rAAV6) Potently and Preferentially Transduces Rat Astrocytes In vitro and In vivo

Gene Therapy Using a miniCEP290 Fragment Delays Photoreceptor Degeneration in a Mouse Model of Leber Congenital Amaurosis

Titration of Lentivirus Vectors

Production of Recombinant Adeno-Associated Viruses (rAAVs) by Transient Transfection

Development and application of an antigen capture ELISA assay for diagnosis of Japanese encephalitis virus in swine, human and mosquito

Enrichment of Fully Packaged Virions in Column-Purified Recombinant Adeno-Associated Virus (rAAV) Preparations by Iodixanol Gradient Centrifugation Followed by Anion-Exchange Column Chromatography

AAV-delivered suppressor tRNA overcomes a nonsense mutation in mice

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