Gene therapy delivered micro-dystrophins co-localize with transgenic utrophin in dystrophic skeletal muscle fibers
S Krishna, AB Piepho, DM Lake, LR Cumby… - Neuromuscular …, 2024 - Elsevier
Duchenne muscular dystrophy (DMD) is a devastating muscle disease caused by the
absence of functional dystrophin. There are multiple ongoing clinical trials for DMD that are
testing gene therapy treatments consisting of adeno-associated viral (AAV) vectors carrying
miniaturized versions of dystrophin optimized for function, termed micro-dystrophins (μDys).
Utrophin, the fetal homolog of dystrophin, has repeatedly been reported to be upregulated in
human DMD muscle as a compensatory mechanism, but whether µDys displaces full-length …
absence of functional dystrophin. There are multiple ongoing clinical trials for DMD that are
testing gene therapy treatments consisting of adeno-associated viral (AAV) vectors carrying
miniaturized versions of dystrophin optimized for function, termed micro-dystrophins (μDys).
Utrophin, the fetal homolog of dystrophin, has repeatedly been reported to be upregulated in
human DMD muscle as a compensatory mechanism, but whether µDys displaces full-length …