BioCentury
ARTICLE | Targets & Mechanisms

Targeting miRNAs in ALS

January 7, 2010 8:00 AM UTC

A team from The University of Texas Southwestern Medical Center at Dallas has identified a muscle-specific microRNA-miR-206-that plays a role in the progression of amyotrophic lateral sclerosis.1 The results suggest that muscle cells could be therapeutic targets in addition to the more commonly pursued motor neurons. miRagen Therapeutics Inc. has licensed the findings and plans to design a therapeutic and eventually seek a partner to test it in the clinic.

ALS is an adult motor neuron disease characterized by atrophy and paralysis of lower limb and respiratory muscles. Nonfamilial, sporadic ALS is the most common form. Familial ALS occurs in about 20% of patients, and about a quarter of those cases are linked to mutations in the gene coding for superoxide dismutase 1 (SOD1). Rilutek riluzole, a sodium channel blocker from sanofi-aventis Group, is the only approved treatment...