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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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CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors

Cell, 2020-04, Vol.181 (1), p.136-150

2020

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Viral vector platforms within the gene therapy landscape

Signal transduction and targeted therapy, 2021-02, Vol.6 (1), p.53-24, Article 53

2021

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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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Regulation of RIPK1 activation by TAK1-mediated phosphorylation dictates apoptosis and necroptosis

Nature communications, 2017-08, Vol.8 (1), p.359-12, Article 359

2017

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Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses

Human gene therapy, 2015-07, Vol.26 (7), p.432

2015

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Rational design of aptazyme riboswitches for efficient control of gene expression in mammalian cells

eLife, 2016-11, Vol.5

2016

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Dynamics of a disinhibitory prefrontal microcircuit in controlling social competition

Neuron (Cambridge, Mass.), 2022-02, Vol.110 (3), p.516-531.e6

2022

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Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

Nature biotechnology, 2016-03, Vol.34 (3), p.328-333

2016

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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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Quantitative and Digital Droplet-Based AAV Genome Titration

Methods in molecular biology (Clifton, N.J.), 2019, Vol.1950, p.51

2019

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Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

Molecular therapy, 2022-08, Vol.30 (8), p.2646-2663

2022

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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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MicroRNA 122, Regulated by GRLH2, Protects Livers of Mice and Patients From Ethanol-Induced Liver Disease

Gastroenterology (New York, N.Y. 1943), 2018-01, Vol.154 (1), p.238-252.e7

2018

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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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LATS suppresses mTORC1 activity to directly coordinate Hippo and mTORC1 pathways in growth control

Nature cell biology, 2020-02, Vol.22 (2), p.246

2020

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Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10

Molecular therapy, 2014-07, Vol.22 (7), p.1299-1309

2014

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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

2018

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Canavan Disease as a Model for Gene Therapy-Mediated Myelin Repair

Frontiers in cellular neuroscience, 2021-04, Vol.15, p.661928-661928

2021

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Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System

Molecular therapy, 2011-08, Vol.19 (8), p.1440-1448

2011

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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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Start codon disruption with CRISPR/Cas9 prevents murine Fuchs' endothelial corneal dystrophy

eLife, 2021-06, Vol.10

2021

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Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity

Molecular therapy, 2017-06, Vol.25 (6), p.1363-1374

2017

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

CRISPR-Based Therapeutic Genome Editing: Strategies and In Vivo Delivery by AAV Vectors

Viral vector platforms within the gene therapy landscape

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

Regulation of RIPK1 activation by TAK1-mediated phosphorylation dictates apoptosis and necroptosis

Adenovirus-Mediated Somatic Genome Editing of Pten by CRISPR/Cas9 in Mouse Liver in Spite of Cas9-Specific Immune Responses

Rational design of aptazyme riboswitches for efficient control of gene expression in mammalian cells

Dynamics of a disinhibitory prefrontal microcircuit in controlling social competition

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

Worldwide Epidemiology of Neutralizing Antibodies to Adeno-Associated Viruses

Quantitative and Digital Droplet-Based AAV Genome Titration

Evaluating the state of the science for adeno-associated virus integration: An integrated perspective

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

MicroRNA 122, Regulated by GRLH2, Protects Livers of Mice and Patients From Ethanol-Induced Liver Disease

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

LATS suppresses mTORC1 activity to directly coordinate Hippo and mTORC1 pathways in growth control

Global CNS Transduction of Adult Mice by Intravenously Delivered rAAVrh.8 and rAAVrh.10 and Nonhuman Primates by rAAVrh.10

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

Canavan Disease as a Model for Gene Therapy-Mediated Myelin Repair

Several rAAV Vectors Efficiently Cross the Blood–brain Barrier and Transduce Neurons and Astrocytes in the Neonatal Mouse Central Nervous System

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

Start codon disruption with CRISPR/Cas9 prevents murine Fuchs' endothelial corneal dystrophy

Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity

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