Source: Cold Spring Harbor Laboratory
Date: April 5, 2008
Summary:
In the neuromuscular disease called spinal muscular atrophy, or SMA, a protein deficiency caused by a single gene mutation leads to serious damage in growing nerve cells and the muscles they control. Now, in laboratory experiments, researchers at Cold Spring Harbor Laboratory (CSHL) and Isis Pharmaceuticals have induced cells to replenish the protein by activating an existing, slightly modified copy of the mutant gene. These early results hold out hope for one day successfully treating this often-fatal disease.
