A review of Genetic Etiology and Emerging Molecular Therapies for FSHD in Preclinical Studies

Abstract

Facioscapulohumeral muscular dystrophy is one of the most common musculoskeletal diseases with a considerable burden. Most of the affected individuals experience muscle weakness as the common muscular symptom. Despite the underlying genetic mechanism which is extensively studied, curative treatment is not available for patients with facioscapulohumeral muscular dystrophy, and only supportive care is considered as the treatment of choice. Recently, several studies addressed the treatment of facioscapulohumeral muscular dystrophy by genetic engineering strategies, most of which indicate the effectiveness of different types of small interfering ribonucleic acids. However, these studies are still in the preclinical phase and it seems that there is a long way ahead of curing facioscapulohumeral muscular dystrophy despite recent advances in the field of genetic engineering. This study aimed to review the underlying genetic mechanism of Facioscapulohumeral muscular dystrophy alongside providing the latest preclinical studies related to the treatment of this disease.

Keywords

• facioscapulohumeral muscular dystrophy
• small interfering rna
• genetic therapy