LENTIVIRAL VECTORS WITH TROPISM TO MOTOR NEURONS COMPRISING AN ANTIBODY THAT BINDS TO A PRE-SYNAPTIC TERMINAL RECEPTOR ON THE NEUROMUSCULAR JUNCTION AND A FUSOGENIC PROTEIN

摘要

The present invention relates to vectors useful in the treatment of disease through targeted delivery. In particular, the present invention relates to vectors useful in the treatment of disease associated with neuronal degeneration, such as amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA).