New Familial ALS Mouse Model Results Promising
Roberta Friedman, PhD, ALSA Research Department Information Coordinator
April 25, 2005
QUICK SUMMARY: A new approach to a therapy for ALS, also referred to as Lou Gehrig’s disease, has added muscle strength and life span in animal models of the disease. The gene silencing approach, called RNA inhibition, prevents a specific gene from making its protein. The target is the enzyme called SOD1. This mutation appears in some cases of inherited ALS. The fact that two separate teams working toward an ALS therapeutic have recently reported similar hopeful findings with RNA silencing strategies shows that the approach indeed has promise for rapid progress into clinical application, said Lucie Bruijn, Ph.D., science director and vice president of The ALS Association (ALSA).
Scientists have created mice that have the mutant SOD1 and also show symptoms remarkably similar to all types of ALS. Mice with the SOD1 mutation show better grip strength if treated with the RNA therapeutic, according to the report online April 25 in the Annals of Neurology, by ALSA funded investigators Timothy Miller M.D. and Don Cleveland, Ph.D., of the Ludwig Institute at the University> of California, San Diego, and collaborators. Rapid application of this technique to ALS investigation is part of ALSA’s program to harness new methods toward changing the disease.
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