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Gene-Silencing Technique Could Fight ALS

In mice, it greatly delayed disease onset

MONDAY, March 14, 2005 (HealthDay News) -- A technique that effectively silences a mutated gene responsible for some cases of amyotrophic lateral sclerosis (ALS) shows promise as a treatment for the fatal motor neuron disease, say French researchers.

In research with mice, the gene technique -- called RNA interference -- greatly delayed the onset and progression of ALS, also known as Lou Gehrig's disease. At the same time, the researchers were able to deliver a normal, healthy version of the gene to motor neuron cells.

"This is the first proof of principle in the human form of a disease of the nervous system in which you can silence the gene and at the same time produce another normal form of the protein," study co-author Patrick Aebischer, president of the Ecole Polytechnique Federale de Lausanne, said in a prepared statement.

"I would not be surprised to see, in the next 10 years, this technology used for treating diseases of the nervous system," he added.

Aebischer and his colleagues targeted mutations in a gene that expresses superoxide dismutase enzyme (SOD1). About 20 percent of the 5 percent to 10 percent of inherited ALS cases have been linked to more than 100 mutations in this SOD1 gene. Most cases of ALS have no identifiable cause, however.

Reporting in the April issue of Nature Medicine, Aebischer's team focused their research on mice bred to express the human SOD1 gene. They successfully used RNA interference to silence the defective gene and prevent it from expressing the SOD1 protein.

About 5,000 Americans are diagnosed each year with ALS, a progressive neurological disease that destroys the motor neurons that control muscles. ALS patients eventually experience gradual paralysis and eventually lose all motor function, leaving them unable to speak, swallow or breathe.

More information

The Muscular Dystrophy Association has more about ALS.

SOURCE: Ecole Polytechnique Federale de Lausanne, news release, March 13, 2005
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