Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models

Song Y, Morales L, Malik AS, et al. Non-immunogenic utrophin gene therapy for the treatment of muscular dystrophy animal models. Nature medicine 2019;25:1505-1511   Abstract The essential product of the Duchenne muscular dystrophy (DMD) gene is dystrophin(1), a rod-like protein(2) that protects striated myocytes from contraction-induced injury(3,4). dystrophin-related protein (or utrophin) retains most of the […]