Fourth causative gene implicates new molecular pathways, potential therapeutic targets

A collaborative research effort spanning nearly a decade between Harvard researchers at Massachusetts General Hospital (MGH) and King’s College London (KCL) has identified a novel gene for inherited amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s disease).

This is the fourth gene associated with familial forms of the devastating neurological disorder. Two papers report mutations in FUS/TLS, a gene known to play a role in DNA repair and the regulation of gene expression.  The mutations affect the behavior of the FUS/TLS protein within cells and lead to deposits of abnormal protein within motor neurons.

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