Gene therapy holds promise for treating inherited Lou Gehrig's Disease

CHICAGO --- Using gene therapy in laboratory mice, researchers have halted motor neuron destruction and slowed progression of inherited amyotrophic lateral sclerosis (ALS), or Lou Gehrig's disease, a lethal, progressive neurological disorder that renders the muscles of the body useless while leaving the mind unaffected.

This finding suggests that a similar gene therapy approach might someday prove effective in treating humans with ALS, according to Northwestern University developmental neurobiologist Martha C. Bohn and colleagues in an article in the July 15 issue of Human Gene Therapy.

Bohn is a professor of pediatrics at Northwestern University Medical School and director of the neurobiology program at Children's Memorial Institute for Education and Research.

Into the hindlimbs of an ALS mouse model, Bohn and co-researchers injected young skeletal muscle cells (myoblasts) infected with a virus that had been made harmless and also engineered to carry the gene for glial cell-derived neurotrophic factor (GDNF). GDNF is a protein that has been found to block nerve cell degeneration in animal models of Parkinson's disease.

The GDNF gene therapy enhanced survival of motor neurons in the laboratory model of inherited ALS and increased the number of motor neurons that maintained 'communication' with cells in treated leg muscles. Additionally, GDNF gene therapy slowed shrinkage of motor neurons and muscle atrophy, improved motor function in affected mice and delayed onset of ALS disease symptoms.

"The most critical issues in designing a gene therapy approach for any human disease are to have a good animal model of the disease and to choose the appropriate target tissue and mode of gene delivery," Bohn said.

The mice used in this study express a mutant gene that is carried by humans with a hereditary form of ALS. These mice were developed by Mark Gurney and Teepu Siddique, professor of neuro

Contact: Elizabeth Crown
Northwestern University

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