DEVELOPMENT OF A NOVEL THERAPY FOR SPINAL MUSCULAR ATROPHY

This proposal is designed to develop a novel therapy for the treatment of a severe form of muscle degeneration called Spinal Muscular Atrophy type I (SMA). One in every 6000 children is born with this disease that causes paralysis. SMA affects motor neuron cells in the spinal cord, which control the body’s voluntary muscles from the head down. In the severe form of SMA, children become paralyzed soon after birth and die from respiratory failure within the first 2 years of life and in the milder juvenile onset forms, affected children are unable to walk. There is currently no treatment available for these patients. We propose to use a nonviral gene therapy approach to fix the defect that causes the disease.