Genetics
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.
The mission of Project A.L.S.™ is to recruit the world’s best research scientists and clinicians to work together toward an understanding of and the first effective treatments for ALS (amyotrophic lateral sclerosis), also known as Lou Gehrig’s disease.
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