Impact Factor (2025): 6.9
DOI Prefix: 10.47001/IRJIET
Muscular
dystrophy, also known as Duchenne muscular dystrophy (DMD), is a fatal muscle
disorder, changes to the DMD gene be the cause of this disease, DMD primarily
impacts boys during childhood. For DMD there was no treatment available up
until, in recent years, a range of curative approaches to Muscular Dystrophy
have been under research; a few among them are development of corticosteroids,
viral vector mediated gene therapy, exon skipping, etc. exon-skipping is the
one of the optimistic approaches for correcting DMD, this therapy’s main aim is
to transform an out-of-frame mutation to convert an out-of-frame mutation into
an in-frame mutation, to change of a severe DMD phenotype into a mild phenotype
by restoration of reduced dystrophin aspect.
Country : India
IRJIET, Volume 8, Issue 8, August 2024 pp. 264-267