Duchenne’s Muscular Dystrophy: The Role of Induced Pluripotent Stem Cells and Genomic Editing on Muscle Regeneration

Abstract

There are two types of well-known muscular dystrophies: Duchenne's muscular dystrophy (DMD) and Becker's muscular dystrophy. This article focuses on the X-linked recessive disorder of Duchenne's muscular dystrophy, which primarily affects children at age four, with a shortened life span of up to 40 years. A defective dystrophin protein lacking the gene dystrophin is the primary cause of the disease pathophysiology. This defect causes cardiac and skeletal muscle down-regulation of dystrophin, leading to weak and fibrotic muscles. The disease is currently untreatable, so most kids die due to cardiac failure in their late 30's. This review presents current treatment options, based on previous studies conducted over the last five years. We used the PubMed database to analyze and review the most important investigations. We also included an analysis of induced pluripotent stem cell therapy vs. genetic therapy using the mdx mouse model. We have discovered promising results on mdx mouse models to date and excited about the potential for where further clinical human trials can go.