Abstract
Duchenne's muscular dystrophy (DMD) is a severe X-linked disorder characterized by
progressive muscle degeneration, leading to loss of ambulation, respiratory failure,
and premature death. It affects approximately 1 in 3,500 live male births and is caused
by mutations in the dystrophin gene, which impairs muscle fiber stability. Current
treatments are limited to managing symptoms and slowing disease progression, with
no curative therapies available. The advent of CRISPR/Cas9 gene-editing technology
has introduced a promising approach for directly correcting the genetic mutations
responsible for DMD. This review explores the potential of CRISPR/Cas9 as a transformative
therapy for DMD, highlighting its successes in preclinical models, the challenges
associated with its delivery, and the obstacles to its clinical application. While
preclinical studies demonstrate the efficacy of CRISPR/Cas9 in restoring dystrophin
expression and improving muscle function, significant hurdles remain, including optimizing
delivery methods and ensuring long-term safety.
Keywords
CRISPR/Cas9 - Duchenne's muscular dystrophy - gene editing - gene therapy - preclinical
models
