A Permanent Genetic Treatment for Spinal Muscular Atrophy Using Base Editors

Explore cutting-edge research on a potential permanent genetic treatment for Spinal Muscular Atrophy (SMA) using base editors in this 35-minute webinar presented by Dr. Christiano R. R. Alves. Delve into the innovative approach of editing the SMN2 gene to restore SMN protein levels, a crucial factor in treating this devastating neuromuscular disease. Learn about the testing of various adenosine base editors (ABEs) and Cas9 variants, resulting in significant intended editing in patient-derived fibroblasts and increased SMN2 exon 7 and SMN protein levels. Discover the potential of a dual adeno-associated virus approach for precise SMN2 editing in an SMA mouse model. Gain insights into the development of ABEs fused with high-fidelity Cas9 variants to reduce off-target editing. Understand how these novel technologies may offer advantages over exogenous gene replacement, potentially providing a long-lasting cure for SMA. PACE credits are available upon completion of the webinar, with an expiration date of May 17, 2025.