Kevin M. Flanigan

Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital

Affiliated withThe Research Institute at Nationwide Children’s Hospital The Ohio State University

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JoVE Journal Publications

ArticleTotal : 1	Year
Publication title Cited by 3	2021

Other Publications

Article	Year
Analysis of dystrophin deletion mutations predicts age of cardiomyopathy onset in becker muscular dystrophy. Circulation. Cardiovascular genetics\| PubMed ID: 20031633	2009
Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene. Human mutation\| PubMed ID: 21972111	2011
RNA interference inhibits DUX4-induced muscle toxicity in vivo: implications for a targeted FSHD therapy. Molecular therapy : the journal of the American Society of Gene Therapy\| PubMed ID: 22508491	2012
The muscular dystrophies. Seminars in neurology\| PubMed ID: 23117950	2012
Correlation of knee strength to functional outcomes in Becker muscular dystrophy. Muscle & nerve\| PubMed ID: 23460325	2013
Proof of concept of the ability of the kinect to quantify upper extremity function in dystrophinopathy. PLoS currents\| PubMed ID: 23516667	2013
Identification of new dystroglycan complexes in skeletal muscle. PloS one\| PubMed ID: 23951345	2013
Anti-dystrophin T cell responses in Duchenne muscular dystrophy: prevalence and a glucocorticoid treatment effect. Human gene therapy\| PubMed ID: 24010700	2013
The ZZ domain of dystrophin in DMD: making sense of missense mutations. Human mutation\| PubMed ID: 24302611	2014
A comparative study of N-glycolylneuraminic acid (Neu5Gc) and cytotoxic T cell (CT) carbohydrate expression in normal and dystrophin-deficient dog and human skeletal muscle. PloS one\| PubMed ID: 24505439	2014
Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates. Human gene therapy. Clinical development\| PubMed ID: 24720466	2014
Duchenne and Becker muscular dystrophies. Neurologic clinics\| PubMed ID: 25037084	2014
Dystrophin quantification: Biological and translational research implications. Neurology\| PubMed ID: 25355828	2014
Reliability and validity of active-seated: An outcome in dystrophinopathy. Muscle & nerve\| PubMed ID: 25641021	2015
The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development. Orphanet journal of rare diseases\| PubMed ID: 25902795	2015
Genetics and emerging treatments for Duchenne and Becker muscular dystrophy. Pediatric clinics of North America\| PubMed ID: 26022172	2015
How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the U.S. Food and Drug Administration. Orphanet journal of rare diseases\| PubMed ID: 26104810	2015
The first exon duplication mouse model of Duchenne muscular dystrophy: A tool for therapeutic development. Neuromuscular disorders : NMD\| PubMed ID: 26365037	2015
Dp412e: a novel human embryonic dystrophin isoform induced by BMP4 in early differentiated cells. Skeletal muscle\| PubMed ID: 26568816	2015
A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB. Human gene therapy. Clinical development\| PubMed ID: 26684447	2015
Duchenne muscular dystrophy: meeting the therapeutic challenge. The Lancet. Neurology\| PubMed ID: 27302348	2016
Modeling functional decline over time in sporadic inclusion body myositis. Muscle & nerve\| PubMed ID: 27511790	2017
A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design. Molecular genetics and metabolism\| PubMed ID: 27590925	2016
N-terminal α Dystroglycan (αDG-N): A Potential Serum Biomarker for Duchenne Muscular Dystrophy. Journal of neuromuscular diseases\| PubMed ID: 27854211	2016
Comparison of Serum rAAV Serotype-Specific Antibodies in Patients with Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Inclusion Body Myositis, or GNE Myopathy. Human gene therapy\| PubMed ID: 28042944	2017
The 100-meter timed test: Normative data in healthy males and comparative pilot outcome data for use in Duchenne muscular dystrophy clinical trials. Neuromuscular disorders : NMD\| PubMed ID: 28279570	2017
Impaired regeneration in calpain-3 null muscle is associated with perturbations in mTORC1 signaling and defective mitochondrial biogenesis. Skeletal muscle\| PubMed ID: 29241457	2017
Low-level dystrophin expression attenuating the dystrophinopathy phenotype. Neuromuscular disorders : NMD\| PubMed ID: 29305136	2018
Long-range genomic regulators of THBS1 and LTBP4 modify disease severity in duchenne muscular dystrophy. Annals of neurology\| PubMed ID: 30014611	2018
An Isolated Limb Infusion Method Allows for Broad Distribution of rAAVrh74.MCK. to Leg Skeletal Muscles in the Rhesus Macaque. Molecular therapy. Methods & clinical development\| PubMed ID: 30073180	2018
Evaluation of biomarkers for Sanfilippo syndrome. Molecular genetics and metabolism\| PubMed ID: 31104888	2019
Lack of toxicity in non-human primates receiving clinically relevant doses of an AAV9.U7snRNA vector designed to induce DMD exon 2 skipping. Human gene therapy\| PubMed ID: 33406986	2021