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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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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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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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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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Significance of perihematomal edema in acute intracerebral hemorrhage: the INTERACT trial

Neurology, 2009-12, Vol.73 (23), p.1963-1968

2009

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Large-scale molecular epidemiological analysis of AAV in a cancer patient population

Oncogene, 2021-04, Vol.40 (17), p.3060-3071

2021

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Genome-wide DNase hypersensitivity, and occupancy of RUNX2 and CTCF reveal a highly dynamic gene regulome during MC3T3 pre-osteoblast differentiation

PloS one, 2017-11, Vol.12 (11), p.e0188056-e0188056

2017

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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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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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Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?

Frontiers in immunology, 2021-05, Vol.12, p.673699-673699

2021

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Additive Effects of Environmental Enrichment and Ketamine on Neuropathic Pain Relief by Reducing Glutamatergic Activation in Spinal Cord Injury in Rats

Frontiers in neuroscience, 2021-03, Vol.15, p.635187

2021

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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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Rational engineering of a functional CpG-free ITR for AAV gene therapy

Gene therapy, 2022-06, Vol.29 (6), p.333-345

2022

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The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2

Gene therapy, 2024-09, Vol.31 (9-10), p.489-498

2024

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Aberrant Expression of a Proliferation-Inducing Ligand Underlies Autoimmune Mechanisms in Immune Thrombocytopenia

Journal of Immunology Research, 2021, Vol.2021, p.3676942-9

2021

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Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice

Nature biotechnology, 2018-10, Vol.36 (9), p.839-842

2018

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Effects of extended in-patient treatment training on outcome of post-stroke dysphagia

European review for medical and pharmacological sciences, 2017-12, Vol.21 (24), p.5711

2017

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Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction

Frontiers in immunology, 2021-04, Vol.12, p.674242-674242

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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Adiponectin regulates thermal nociception in a mouse model of neuropathic pain

British journal of anaesthesia : BJA, 2018-06, Vol.120 (6), p.1356-1367

2018

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Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor

EMBO molecular medicine, 2024-04, Vol.16 (4), p.945-965

2024

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High-Throughput Quantification of In Vivo Adeno-Associated Virus Transduction with Barcoded Non-Coding RNAs

Human gene therapy, 2019-08, Vol.30 (8), p.946-956

2019

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Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras

Molecular therapy. Methods & clinical development, 2018-06, Vol.9, p.130-141

2018

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Thyroid Hormone Receptor-β (TRβ) Mediates Runt-Related Transcription Factor 2 (Runx2) Expression in Thyroid Cancer Cells: A Novel Signaling Pathway in Thyroid Cancer

Endocrinology (Philadelphia), 2016-08, Vol.157 (8), p.3278-3292

2016

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

Viral vector platforms within the gene therapy landscape

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

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

Significance of perihematomal edema in acute intracerebral hemorrhage: the INTERACT trial

Large-scale molecular epidemiological analysis of AAV in a cancer patient population

Genome-wide DNase hypersensitivity, and occupancy of RUNX2 and CTCF reveal a highly dynamic gene regulome during MC3T3 pre-osteoblast differentiation

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

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

Vectored Immunotherapeutics for Infectious Diseases: Can rAAVs Be The Game Changers for Fighting Transmissible Pathogens?

Additive Effects of Environmental Enrichment and Ketamine on Neuropathic Pain Relief by Reducing Glutamatergic Activation in Spinal Cord Injury in Rats

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

Rational engineering of a functional CpG-free ITR for AAV gene therapy

The AAV2.7m8 capsid packages a higher degree of heterogeneous vector genomes than AAV2

AAV gene therapy for Tay-Sachs disease

Aberrant Expression of a Proliferation-Inducing Ligand Underlies Autoimmune Mechanisms in Immune Thrombocytopenia

Cas9-mediated allelic exchange repairs compound heterozygous recessive mutations in mice

Effects of extended in-patient treatment training on outcome of post-stroke dysphagia

Novel Combinatorial MicroRNA-Binding Sites in AAV Vectors Synergistically Diminish Antigen Presentation and Transgene Immunity for Efficient and Stable Transduction

Short DNA Hairpins Compromise Recombinant Adeno-Associated Virus Genome Homogeneity

Adiponectin regulates thermal nociception in a mouse model of neuropathic pain

Improved gene therapy for spinal muscular atrophy in mice using codon-optimized hSMN1 transgene and hSMN1 gene-derived promotor

High-Throughput Quantification of In Vivo Adeno-Associated Virus Transduction with Barcoded Non-Coding RNAs

Adeno-associated Virus Genome Population Sequencing Achieves Full Vector Genome Resolution and Reveals Human-Vector Chimeras

Thyroid Hormone Receptor-β (TRβ) Mediates Runt-Related Transcription Factor 2 (Runx2) Expression in Thyroid Cancer Cells: A Novel Signaling Pathway in Thyroid Cancer

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