SMN2 genes

Scientists use a small molecule to coax synthesis of a needed protein in SMA-affected cells

posted on July 31, 2009 - 10:25am
Scientists at three U.S. institutions have used a very small synthetic molecule to correct the genetic defect in cells taken from a person with spinal muscular atrophy (SMA), a disease in which muscle-controlling nerve cells in the spinal cord are lost.
posted on April 1, 2009 - 12:26pm
QUEST Vol. 16, No. 2
Story includes research items about: Charcot-Marie-Tooth disease, congenital muscular dystrophy, Duchenne muscular dystrophy, Emery-Dreifuss muscular dystrophy (EDMD), type 1 myotonic muscular dystrophy, and spinal muscular atrophy - including, type 3 SMA and spinal-bulbar muscular atrophy (SBMA, Kennedy disease).