Amyotrophic lateral sclerosis and gene therapy.

As our understanding of the molecular basis of neurodegenerative diseases continues to improve, the prospect of using gene therapy to treat these conditions becomes increasingly realistic. This Viewpoint article discusses initial trials of an RNA interference gene-silencing strategy to modify the expression of mutant proteins in animal models of amyotrophic lateral sclerosis, and considers the hurdles that will need to be overcome to translate this approach into the clinic.