CRISPR/Cas9 Gene Therapy for Spinal Muscular Atrophy: Promising Preclinical Data
CRISPR/Cas9 gene therapy holds promising potential for treating spinal muscular atrophy (AME). Preclinical studies have demonstrated its ability to restore SMN protein levels and improve motor function in animal models of SMA. These findings suggest that CRISPR/Cas9 could be a transformative therapeutic approach for this debilitating disease.