An appraisal of emerging dystrophin restoration therapies in Duchenne muscular dystrophy

Abstract

Duchenne muscular dystrophy (DMD) is an X-linked, progressive muscle disorder caused by pathogenic variants in the DMD gene and resulting in a complete loss of dystrophin protein expression. As of now, there is no cure for DMD, and despite improvements in standard of care, there are significant unmet needs for disease modifying treatments. This article provides an overview of emerging therapies aimed at dystrophin restoration, emphasizing exon skipping and gene therapy, within the rapidly evolving landscape for Duchenne muscular dystrophy.

Keywords

Duchenne muscular dystrophy / dystrophin / gene therapy / exon skipping

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