The potential role of miRNA therapies in spinal muscle atrophy

Spinal muscular atrophy (SMA) is a neurodegenerative disease caused by low levels of full-length survival motor neuron (SMN) protein due to the loss 1 (SMN1) gene and inefficient splicing 2 (SMN2) gene, which mostly affects alpha neurons lower spinal cord. Despite U.S. Food Drug Administration (FDA) approved SMN-dependent therapies including Nusinersen, Zolgensma® Evrysdi™, SMA still devastating as these existing expensive drugs may not be sufficient thus, remains need for additional therapies. The involvement microRNAs (miRNAs) in expanding because miRNAs are important mediators expression each miRNA could target number genes. Hence, miRNA-based therapy utilized treating this genetic disorder. However, delivery into cells an obstacle SMA, there no effective system date. This review highlights potential strategies intracellular current challenges delivery. Furthermore, we provide future prospects therapeutic SMA.