Genetic strategies for therapy of Duchenne muscular dystrophy.

ABSTRACT: Duchenne muscular dystrophy (DMD) is a severe, X-linked genetic disorder caused by mutations in the DMD gene, which encodes dystrophin, an essential structural muscle protein. Currently, there are no cures for DMD, and available therapies primarily focus on alleviating symptoms rather than correcting the underlying genetic defect. However, restoration of a shortened version of dystrophin offers the potential for partially addressing the underlying cause of the disease. This review focuses on the promises and challenges of various genetic strategies, such as exon skipping, gene replacement, and gene editing (e.g., by CRISPR-Cas9) aimed at restoring or replacing the dystrophin expression or upregulating utrophin, a paralog of dystrophin that is primarily expressed during fetal life. F

SUBMITTER: Loboda A 

PROVIDER: S-EPMC12664987 | biostudies-literature | 2025 Dec

REPOSITORIES: biostudies-literature