COLUMBIA, Mo.-- Usually, results from a new study help
scientists inch their way toward an answer whether they are
battling a health problem or are on the verge of a technological
breakthrough. Once in a while, those results give them a giant
leap forward. In a preliminary study in a canine model of
Duchenne muscular dystrophy (DMD), University of Missouri
scientists showed exactly such a leap using gene therapy to
treat muscular dystrophy. The results of the study will be
published in the journal Molecular Therapy on Jan. 15, 2013.
Muscular dystrophy occurs when damaged muscle tissue is replaced
with fibrous, bony or fatty tissue and loses function. Duchenne
muscular dystrophy is the most common type of muscular dystrophy
predominantly affecting boys. Patients with DMD have a gene
mutation that disrupts the production of dystrophin, a protein
essential for muscle cell survival and function. Absence of
dystrophin starts a chain reaction that eventually leads to
muscle cell degeneration and death. For years, s