Use of an AAV9-based system for vectored immunoprophylaxis (VIP) targeting SOD1 in amyotrophic lateral sclerosis (ALS). KU Leuven
Amyotrophic lateral sclerosis (ALS) is an adult-onset neurodegenerative disease that is almost always fatal. In 20% of familial ALS patients, ALS is caused by mutations in the superoxide dismutase 1 (SOD1) gene. These mutations alter the conformation of SOD1 and it starts to form aggregates. It is thought that misfolded SOD1 is toxic, causing motor neuron death. One possible way to treat ALS is to reduce the levels of toxic SOD1. Antibodies ...