Pre-clinical developments in neuromuscular disorders

The field of gene therapy for Duchenne muscular dystrophy (DMD) has been very active in the past few years with multiple clinical trials going on. The large size of the disease causal gene DMD presents a challenge for AAV gene therapy as the cDNA of dystrophin is 14 kb, well over the carrying capacity of AAV. Thus, different AAV-microdystrophins (truncated forms of dystrophin) has been the main form of gene therapy for DMD in clinical development. While the trials are showing promising results, the limited transgene size and threat of anti-dystrophin immune response indicate the necessity to explore other strategies.

Source: Neuromuscular Disorders - September 19, 2021 Category: Neurology Authors: Y. Zhang, M. Lambert, J. Widrick, J. Conner, J. Spinazzola, L. Kunkel Source Type: research