Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy

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Very mild muscular dystrophy associated with the deletion of 46% of dystrophin

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Viral vector platforms within the gene therapy landscape

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In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy

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Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy

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Assessment of Systemic Delivery of rAAVrh74.MHCK7.micro-dystrophin in Children With Duchenne Muscular Dystrophy: A Nonrandomized Controlled Trial

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Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy

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

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Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors

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Adeno-Associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury

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Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery

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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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A hybrid vector system expands adeno-associated viral vector packaging capacity in a transgene-independent manner

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Treatment of human disease by adeno-associated viral gene transfer

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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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Systemic Microdystrophin Gene Delivery Improves Skeletal Muscle Structure and Function in Old Dystrophic mdx Mice

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AAV.Dysferlin Overlap Vectors Restore Function in Dysferlinopathy Animal Models

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Gene Therapy of mdx Mice With Large Truncated Dystrophins Generated by Recombination Using rAAV6

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Novel Mini–Dystrophin Gene Dual Adeno-Associated Virus Vectors Restore Neuronal Nitric Oxide Synthase Expression at the Sarcolemma

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

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Full-Length Dystrophin Reconstitution with Adeno-Associated Viral Vectors

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Homologous Recombination Mediates Functional Recovery of Dysferlin Deficiency following AAV5 Gene Transfer

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Can Adeno-Associated Viral Vectors Deliver Effectively Large Genes?

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Dual AAV therapy ameliorates exercise-induced muscle injury and functional ischemia in murine models of Duchenne muscular dystrophy

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

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Trans-splicing adeno-associated viral vector-mediated gene therapy is limited by the accumulation of spliced mRNA but not by dual vector coinfection efficiency

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Dual-AAV delivery of large gene sequences to the inner ear

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Induction of T-Cell Infiltration and Programmed Death Ligand 2 Expression by Adeno-Associated Virus in Rhesus Macaque Skeletal Muscle and Modulation by Prednisone

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Surrogate gene therapy for muscular dystrophy

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