Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular DisordersCamila F. Almeida 1, Emma C. Frair 1, Nianyuan Huang 1, Reid Neinast 2, Kim L. McBride 2,3,4,6, Robert B. Weiss 5, Kevin M. Flanigan 1,6, Nicolas Wein 1,6
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
This protocol describes the conversion of skin fibroblasts into myoblasts and their differentiation into myotubes. The cell lines are derived from patients with neuromuscular disorders and can be used to investigate pathological mechanisms and to test therapeutic strategies.
