Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart

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Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8

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Multiplex CRISPR/Cas9-based genome editing for correction of dystrophin mutations that cause Duchenne muscular dystrophy

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Recombinant Adeno-Associated Virus Serotype 9 Leads to Preferential Cardiac Transduction In Vivo

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Long-term microdystrophin gene therapy is effective in a canine model of Duchenne muscular dystrophy

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Basic Biology of Adeno-Associated Virus (AAV) Vectors Used in Gene Therapy

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A 10 Patient Case Report on the Impact of Plasmapheresis Upon Neutralizing Factors Against Adeno-associated Virus (AAV) Types 1, 2, 6, and 8

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Efficient Neuronal Gene Transfer with AAV8 Leads to Neurotoxic Levels of Tau or Green Fluorescent Proteins

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DNA Shuffling of Adeno-associated Virus Yields Functionally Diverse Viral Progeny

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Molecular evolution of adeno-associated virus for enhanced glial gene delivery

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A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs

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Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver

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Tailoring the AAV vector capsid for gene therapy

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Treatment of human disease by adeno-associated viral gene transfer

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Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency

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Adeno-Associated Virus Biology

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Codon and mRNA sequence optimization of microdystrophin transgenes improves expression and physiological outcome in dystrophic mdx mice following AAV2/8 gene transfer

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Triple Trans-Splicing Adeno-Associated Virus Vectors Capable of Transferring the Coding Sequence for Full-Length Dystrophin Protein into Dystrophic Mice

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Amelioration of laminin-α2-deficient congenital muscular dystrophy by somatic gene transfer of miniagrin

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Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: Current Status of Therapeutic Approaches

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Cardiac-targeted RNA interference mediated by an AAV9 vector improves cardiac function in coxsackievirus B3 cardiomyopathy

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Viral serotype and the transgene sequence influence overlapping adeno‐associated viral (AAV) vector‐mediated gene transfer in skeletal muscle

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Transient Immunomodulation Allows Repeated Injections of AAV1 and Correction of Muscular Dystrophy in Multiple Muscles

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Circumventing cellular immunity by miR142-mediated regulation sufficiently supports rAAV-delivered OVA expression without activating humoral immunity

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Targeted Modifications in Adeno-Associated Virus Serotype 8 Capsid Improves Its Hepatic Gene Transfer Efficiency In Vivo

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

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Enhanced Gene Transfer Efficiency in the Murine Striatum and an Orthotopic Glioblastoma Tumor Model, Using AAV-7- and AAV-8-Pseudotyped Vectors

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AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence

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The AAV-mediated and RNA-guided CRISPR/Cas9 system for gene therapy of DMD and BMD

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Quantitative 3D Tracing of Gene-delivery Viral Vectors in Human Cells and Animal Tissues

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Long-term robust myocardial transduction of the dog heart from a peripheral vein by adeno-associated virus serotype-8

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Inhibition of antigen presentation during AAV gene therapy using virus peptides

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