Treating Duchenne’s Muscular Dystrophy by Using Gene Editing and Gene Therapy

Abstract

Duchenne’s Muscular Dystrophy, DMD, occurs mainly in boys aged two to four and is caused by a genetic mutation that lacks dystrophin, which is a protein that keeps the muscles intact. This paper explores and summarizes several successful gene editing and gene therapy clinical trials that have treated patients with DMD. Using a CRISPR-Cas9 method, it cuts out sections of the dystrophin gene allowing for the cell to make a shortened but functional version of the protein. FDA-approved treatments like Elevidys are a result of gene therapy and it delivers a shortened version of the dystrophin gene in an effort to restore its functionality. While gene editing and gene therapy are a great way to treat DMD and other diseases, there are still many safety and ethical concerns of using these technologies. Ranging from off-target effects, unwanted immune responses, and several ethical implications regarding these technologies. Addressing the many safety and ethical implications of gene editing and gene therapy is a crucial step towards advancing the potential of a future cure for DMD.