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Kevin M. Flanigan

Nationwide Children's Hospital
Ohio State University
University of Utah

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Gene editing and modulation for Duchenne muscular dystrophy

2021
- Anthony A. Stephenson
- Kevin M. Flanigan
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Lack of toxicity in non-human primates receiving clinically relevant doses of an AAV9.U7snRNA vector designed to induce DMD exon 2 skipping.

2021 Human Gene Therapy
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Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy.

2021 Journal of Comparative Effectiveness Research
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Absence of significant off-target splicing variation with a U7snRNA vector targeting DMD exon 2 duplications.

2021 Human Gene Therapy
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Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders

2021 Journal of Visualized Experiments
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Pre-Clinical Dose Escalation Studies Establish a Therapeutic Range for U7snRNA-Mediated DMD Exon 2 Skipping

2021 Molecular therapy. Methods & clinical development
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A genome-wide association analysis of loss of ambulation in dystrophinopathy patients suggests multiple candidate modifiers of disease severity

2021 medRxiv
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Automated immunofluorescence analysis for sensitive and precise dystrophin quantification in muscle biopsies.

2021 Neuropathology and Applied Neurobiology
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The genotypic and phenotypic spectrum of BICD2 variants in spinal muscular atrophy

2020 Annals of Neurology
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Safety, tolerability and preliminary evidence of biopotency in Transpher B, a multicenter, single-dose, phase 1/2 clinical trial of ABO-101 gene therapy for Sanfilippo syndrome type B (mucopolysaccharidosis type IIIB)

2020 Molecular Genetics and Metabolism
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