A person's genetic make-up affects his or her susceptibility to ALS.
Approximately 5% of ALS cases are inherited and are known as familial ALS. Familial ALS is due to a mutation of the gene superoxide dismutase-1 (SOD1) or other genes.
However, most ALS cases do not appear to run in families, and are known as sporadic ALS. In sporadic ALS, a constellation of normally occurring gene variants acts in concert with environmental triggers to cause the disease.
Project A.L.S.™ scientists are working to identify the genetic underpinnings of sporadic ALS. In 2009, Project A.L.S.™ and the Harvard Neurodiscovery Center completed the most comprehensive analysis to date of DNA from ALS patients. Information from that study is guiding the development of new treatments.
Project A.L.S.™ scientists are developing tools to alter gene expression and “turn off” mutated genes in ALS. These tools include RNA interference, zinc fingers, and gene therapy.