Center for Gene Therapy
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Analysis of dystrophin deletion mutations predicts age of cardiomyopathy onset in becker muscular dystrophy.
Circulation. Cardiovascular genetics Dec, 2009 | Pubmed ID: 20031633
Nonsense mutation-associated Becker muscular dystrophy: interplay between exon definition and splicing regulatory elements within the DMD gene.
Human mutation Mar, 2011 | Pubmed ID: 21972111
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 Jul, 2012 | Pubmed ID: 22508491
The muscular dystrophies.
Seminars in neurology Jul, 2012 | Pubmed ID: 23117950
Correlation of knee strength to functional outcomes in Becker muscular dystrophy.
Muscle & nerve Apr, 2013 | Pubmed ID: 23460325
Proof of concept of the ability of the kinect to quantify upper extremity function in dystrophinopathy.
PLoS currents Mar, 2013 | Pubmed ID: 23516667
Identification of new dystroglycan complexes in skeletal muscle.
PloS one , 2013 | Pubmed ID: 23951345
Anti-dystrophin T cell responses in Duchenne muscular dystrophy: prevalence and a glucocorticoid treatment effect.
Human gene therapy Sep, 2013 | Pubmed ID: 24010700
The ZZ domain of dystrophin in DMD: making sense of missense mutations.
Human mutation Feb, 2014 | Pubmed ID: 24302611
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 , 2014 | Pubmed ID: 24505439
Feasibility and safety of systemic rAAV9-hNAGLU delivery for treating mucopolysaccharidosis IIIB: toxicology, biodistribution, and immunological assessments in primates.
Human gene therapy. Clinical development Jun, 2014 | Pubmed ID: 24720466
Duchenne and Becker muscular dystrophies.
Neurologic clinics Aug, 2014 | Pubmed ID: 25037084
Dystrophin quantification: Biological and translational research implications.
Neurology Nov, 2014 | Pubmed ID: 25355828
Reliability and validity of active-seated: An outcome in dystrophinopathy.
Muscle & nerve Sep, 2015 | Pubmed ID: 25641021
The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development.
Orphanet journal of rare diseases Apr, 2015 | Pubmed ID: 25902795
Genetics and emerging treatments for Duchenne and Becker muscular dystrophy.
Pediatric clinics of North America Jun, 2015 | Pubmed ID: 26022172
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 Jun, 2015 | Pubmed ID: 26104810
The first exon duplication mouse model of Duchenne muscular dystrophy: A tool for therapeutic development.
Neuromuscular disorders : NMD Nov, 2015 | Pubmed ID: 26365037
Dp412e: a novel human embryonic dystrophin isoform induced by BMP4 in early differentiated cells.
Skeletal muscle , 2015 | Pubmed ID: 26568816
A GLP-Compliant Toxicology and Biodistribution Study: Systemic Delivery of an rAAV9 Vector for the Treatment of Mucopolysaccharidosis IIIB.
Human gene therapy. Clinical development Dec, 2015 | Pubmed ID: 26684447
Duchenne muscular dystrophy: meeting the therapeutic challenge.
The Lancet. Neurology 07, 2016 | Pubmed ID: 27302348
Modeling functional decline over time in sporadic inclusion body myositis.
Muscle & nerve 04, 2017 | Pubmed ID: 27511790
A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design.
Molecular genetics and metabolism 11, 2016 | Pubmed ID: 27590925
N-terminal α Dystroglycan (αDG-N): A Potential Serum Biomarker for Duchenne Muscular Dystrophy.
Journal of neuromuscular diseases 05, 2016 | Pubmed ID: 27854211
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 09, 2017 | Pubmed ID: 28042944
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 May, 2017 | Pubmed ID: 28279570
Impaired regeneration in calpain-3 null muscle is associated with perturbations in mTORC1 signaling and defective mitochondrial biogenesis.
Skeletal muscle 12, 2017 | Pubmed ID: 29241457
Low-level dystrophin expression attenuating the dystrophinopathy phenotype.
Neuromuscular disorders : NMD 02, 2018 | Pubmed ID: 29305136
Long-range genomic regulators of THBS1 and LTBP4 modify disease severity in duchenne muscular dystrophy.
Annals of neurology 08, 2018 | Pubmed ID: 30014611
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 Sep, 2018 | Pubmed ID: 30073180
Evaluation of biomarkers for Sanfilippo syndrome.
Molecular genetics and metabolism Sep - Oct, 2019 | Pubmed ID: 31104888
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 Jan, 2021 | Pubmed ID: 33406986
Camila F. Almeida1,
Emma C. Frair1,
Nianyuan Huang1,
Reid Neinast2,
Kim L. McBride2,3,4,6,
Robert B. Weiss5,
Kevin M. Flanigan1,6,
Nicolas Wein1,6
1Center for Gene Therapy, The Research Institute at Nationwide Children’s Hospital,
2Center for Cardiovascular Research, The Research Institute at Nationwide Children’s Hospital,
3The Heart Center, Nationwide Children’s Hospital,
4Division of Genetic and Genomic Medicine, Nationwide Children’s Hospital,
5Department of Human Genetics, The University of Utah School of Medicine,
6Department of Pediatrics, The Ohio State University
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