RNA Therapy in Muscle Diseases

Skeletal muscle diseases pose a serious threat to human health, yet effective pharmacological treatments remain scarce. RNA-based therapeutics are emerging as a promising avenue: an RNA drug that restores muscle function has already been approved and is in clinical use, highlighting the potential of RNA therapy in this approach. This review surveys the current landscape of RNA therapies for muscle disorders, focusing on the correction of aberrant messenger RNA (mRNA) metabolism, RNA modifications, and the therapeutic manipulation of noncoding RNAs in skeletal muscle diseases. We outline RNA-directed strategies in skeletal muscle, identify the advantages and challenges of RNA therapy in muscle diseases, and discuss future directions for the field.