CONTACT: L. E. OHMAN
283 Medical Laboratories
Iowa City IA 52242
(319) 335-6660; fax (319) 335-8034
UI researcher receives $300,000 grant to study gene therapy
IOWA CITY, Iowa -- The Muscular Dystrophy Association (MDA) this month awarded
more than $300,000 for the study of gene therapy for congenital muscular dystrophy
(CMD) to Dr. Kevin P. Campbell, University of Iowa professor of physiology
and biophysics and Howard Hughes Medical Institute Investigator.
The award makes research funds available over a three-year period for the
investigation of a novel form of gene therapy to treat CMD in a mouse model
of the disease.
"The goal of this research is to test the feasibility of our gene transfer
approach. Hopefully this will lead to the development of a therapeutic strategy
to treat CMD patients with in the future," Campbell says.
Congenital muscular dystrophy is a group of severe muscle disorders that
differ from Duchenne muscular dystrophy in that the onset of muscle weakness
is present from birth or early infancy. Congenital muscular dystrophy is inherited
by both males and females and has no effective treatment. Several years ago,
Campbell and colleagues showed that a protein that surrounds muscle cells
was absent in CMD patients, and recently defects in the gene for this protein
Another goal of Campbell's research is to better understand the pathogenesis
of this disease. He and his colleagues propose to explore the molecular basis
of this disease in order to determine whether it primarily affects muscle
development or also affects the nerves that control muscle contraction.