Kevin M. Flanigan

Nationwide Children's Hospital — United States

Biochemistry, Genetics and Molecular Biology · Life Sciences

68h-index15.7kcitations347works2.92yr avg
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Research Topics

Muscle Physiology and Disorders(215), Neurogenetic and Muscular Disorders Research(62), RNA Research and Splicing(52), Cardiomyopathy and Myosin Studies(44), Virus-based gene therapy research(30)

Publications346 total

ALT/CK Ratio as an Early Marker of Liver Injury After Gene Therapy in Duchenne Muscular Dystrophy
PEDIATRICS·2026
Five-year follow-up after gene therapy with ABO-101 in five children with Sanfilippo syndrome type B
Molecular Genetics and Metabolism·2026
Predicting subtypes of glycogen storage disease type IV: Challenges of hepatic subtypes and genotype-phenotype correlation
Molecular Genetics and Metabolism·2025
Circulating protein biomarkers identified in two independent clinical trial cohorts of glucocorticoid-naive Duchenne muscular dystrophy patients.
Scientific Reports·2025· 1 citedOpen Access
Re-evaluating acceptable risk of death from gene therapy: A threshold study among individuals with Duchenne muscular dystrophy and their caregivers in the US and UK
European Journal of Medical Genetics·2025Open Access
283PGenomic rearrangements in Duchenne muscular dystrophy diagnosed by RNA and long-read genome sequencing
Neuromuscular Disorders·2025
Consensus recommendations and considerations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy
Neuromuscular Disorders·2025· 3 cited
Like father, like son: RNA-sequencing from a 30-year-old muscle biopsy identifies a novel splice variant in ACTA1 as the cause of an attenuated nemaline myopathy phenotype
Neuromuscular Disorders·2025
Unraveling the spatial landscape of Dystrophinopathies: a transcriptomic approach to Becker and Duchenne muscular dystrophies
2025Open Access
Current clinical applications of AAV-mediated gene therapy
Molecular Therapy·2025· 35 citedOpen Access
Correction of Duchenne Muscular Dystrophy Mutations by CRISPR/Cas9 Gene Editing Using Homology-Independent Targeted Integration (P7-6.006)
Neurology·2025
Treatment with UX111 Gene Therapy Rapidly Reduced Heparan Sulfate (HS) Exposure in Cerebrospinal Fluid (CSF) and Improved Long-term Cognitive Function in Children with Mucopolysaccharidosis IIIA (MPS IIIA) (S31.001)
Neurology·2025· 2 cited
Safety and Efficacy of DYNE-251 in Males with DMD Mutations Amenable to Exon 51 Skipping (S31.010)
Neurology·2025
Generation of an induced pluripotent stem cell line (NCHi023-A) from a 41-year-old male with nemaline myopathy carrying autosomal dominant ACTA1 c.809-10C>A mutation
Stem Cell Research·2025Open Access
Heterozygous loss-of-function variants in SPTAN1 cause an early childhood onset distal myopathy
Genetics in Medicine·2025· 4 cited
Treatment with UX111 gene therapy rapidly reduced heparan sulfate (HS) exposure in cerebrospinal fluid (CSF) and improved long-term cognitive function in children with mucopolysaccharidosis type IIIA (MPS IIIA)
Molecular Genetics and Metabolism·2025
Identification of disease-specific extracellular vesicle-associated plasma protein biomarkers for Duchenne Muscular Dystrophy and Facioscapulohumeral Muscular Dystrophy
2024· 2 citedOpen Access
Gross motor delays in infants and young boys with Duchenne muscular dystrophy
Journal of Neuromuscular Diseases·2024Open Access
435P Correction of a common Duchenne muscular dystrophy mutation by CRISPR/Cas9 gene editing using homology-independent targeted integration
Neuromuscular Disorders·2024
422P U7snRNA-mediated exon 17 skipping restores dystrophin expression in cells and in a novel mouse model of Duchenne muscular dystrophy
Neuromuscular Disorders·2024
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Frequent Co-authors

Jerry R. Mendell(18), Robert B. Weiss(10), Diane M. Dunn(8), Richard S. Finkel(8), Michael Howard(8), Kate Bushby(7), Julaine Florence(7), Louise R. Rodino‐Klapac(6), Craig Campbell(6), Janbernd Kirschner(6), Alan Pestronk(6), Craig M. McDonald(6), Brenda Wong(6), Zarife Sahenk(5), Linda Lowes(5), Lindsay N. Alfano(5), Sarah Lewis(5), Volker Straub(5), Richard J. Barohn(5), Michelle Eagle(5)