Genome Editing-Mediated Utrophin Upregulation in Duchenne Muscular Dystrophy Stem Cells
Kasturi Sengupta, Manoj K. Mishra, Emanuele Loro, Melissa J. Spencer, April D. Pyle, Tejvir S. Khurana
Abstract
genome editing offers a novel utrophin upregulation therapeutic strategy applicable to all DMD patients, irrespective of the dystrophin mutation status.
Topics & Concepts
UtrophinDuchenne muscular dystrophyDystrophinDownregulation and upregulationmicroRNACRISPRUntranslated regionBiologyCell biologyMolecular biologyGeneticsMessenger RNAGeneMuscle Physiology and DisordersCRISPR and Genetic EngineeringVirus-based gene therapy research