Late breaking news e-poster presentation

Duchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene. Although DMD is a progressive degenerative disease, there is evidence for early, embryonic-stage defects in myogenesis and gene expression in DMD. By understanding how these defects initiate and contribute to DMD pathology, we may be better positioned to identify and utilize DMD therapies. Using single-cell RNAseq, we have identified one of the earliest known DMD phenotypes: a novel transcriptional trajectory of DMD human induced pluripotent stem cells (hiPSCs) undergoing myogenesis.

Source: Neuromuscular Disorders - September 28, 2020 Category: Neurology Authors: Dr. Lisa Maves, Jean-Baptiste Dupont, Gist H. Farr, Shawn Luttrell, Melanie Morris, Arianna Gomez, Thao Pham, David Mack Source Type: research