Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy

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UDiTaS™, a genome editing detection method for indels and genome rearrangements

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Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing

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Efficient In Vivo Liver-Directed Gene Editing Using CRISPR/Cas9

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Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

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Progress toward Gene Therapy for Duchenne Muscular Dystrophy

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Genome engineering: a new approach to gene therapy for neuromuscular disorders

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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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Gene therapies that restore dystrophin expression for the treatment of Duchenne muscular dystrophy

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CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice

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Recombinant AAV Integration Is Not Associated With Hepatic Genotoxicity in Nonhuman Primates and Patients

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Analyzing CRISPR genome-editing experiments with CRISPResso

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A multifunctional AAV–CRISPR–Cas9 and its host response

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Engineering Delivery Vehicles for Genome Editing

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Efficient Restoration of the Dystrophin Gene Reading Frame and Protein Structure in DMD Myoblasts Using the CinDel Method

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Viral Vectors for Gene Therapy: Translational and Clinical Outlook

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In vivo genome editing using Staphylococcus aureus Cas9

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Therapeutic genome editing: prospects and challenges

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In vivo gene editing in dystrophic mouse muscle and muscle stem cells

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

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Promoterless gene targeting without nucleases ameliorates haemophilia B in mice

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Engineering adeno-associated viruses for clinical gene therapy

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Duchenne and Becker Muscular Dystrophies

(2014) Kevin M. Flanigan   NEUROLOGIC CLINICS

Intravenous Injections in Neonatal Mice

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The use of self-adjuvanting nanofiber vaccines to elicit high-affinity B cell responses to peptide antigens without inflammation

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Circular RNAs are a large class of animal RNAs with regulatory potency

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AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice

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Immunodominant Liver-Specific Expression Suppresses Transgene-Directed Immune Responses in Murine Pompe Disease

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Long-term Survival of the Juvenile Lethal Arginase-deficient Mouse With AAV Gene Therapy

(2012) Eun K. Lee et al.   MOLECULAR THERAPY

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

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