Dr. Melissa Spencer provides update on development of an LGMD2A/R1 gene therapy
Melissa Spencer, PhD, Chair of the C3 Scientific Advisory Board and Professor of Neurology at UCLA, recently presented during a webinar hosted by the Muscular Dystrophy Association (MDA). The presentation, aimed at educating the public, gave an excellent background about genes, mutations, and adeno-associated vector (AAV)-based gene therapies. She highlights some of the latest advances in gene therapy technologies and provides an update on her program developing an AAV-based gene therapy for LGMD2A/R1. This project received funding from C3, the MDA, and the National Institutes of Health.
Read more about Dr. Spencer’s C3-funded gene therapy program here and MDA here
Advancing Excellence in LGMD Diagnosis, Care, and Emerging Therapies (CME Accreditation) 📅 13-14 November 2026 | 📍 Atlanta, Georgia As new therapies move closer to clinical practice, the need for accurate diagnosis, specialist knowledge, and coordinated multidisciplinary care has never been greater. Developed for healthcare professionals involved in the diagnosis and management of limb-girdle muscular... Read More
A new paper, “CaMKIIβ Signaling drives expression of metabolic and stress response genes in skeletal muscle, and its loss contributes to the LGMDR1 phenotype,” was recently published in the journal Human Molecular Genetics. It was authored by a multi-institutional group led by Dr. Melissa Spencer, C3 Scientific Advisory Board Chair and Professor of Neurology and... Read More