Brain & developmentMuscular DystrophyDuchenneHumansExonsGenetic Therapy
Development and future prospects of exon-skipping therapy for Duchenne muscular dystrophy.
Yasuhiro Takeshima, Tomoko Lee, Hideki Shimomura
Published: 202510.1016/j.braindev.2025.104457
Abstract
Duchenne muscular dystrophy (DMD) is an inherited progressive muscle disease that is caused by variants in the DMD gene. The development of therapies for DMD that promote dystrophin protein production or ameliorate dystrophin deficiency-induced patho…
Preview only. Read the full abstract at the source