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Lab studies show two proteins prevented progressive nerve cell loss in Parkinson's disease

LOS ANGELES (September 8, 2004) In recent years, scientists have made important strides in developing drugs that help patients manage the symptoms of Parkinson's Disease a chronic, progressive movement disorder affecting as many as one million Americans. But despite their effectiveness, the drugs don't stop Parkinson's disease from progressing, causing patients' symptoms to eventually grow worse in spite of medication.

Now, researchers at Cedars-Sinai Medical Center have found that two specific proteins "Sonic Hedgehog" and "Gli-1" delivered via a genetically engineered virus into the brains of laboratory rats, prevented the progressive degeneration of nerve cells in the brain that cause Parkinson's disease. The study, published in the September issue of the journal, Molecular Therapy, may lead to a new way to treat patients with advanced Parkinson's Disease.

"Our results establish, for the first time, that viral transfer of Sonic hedgehog and Gli-1 - two proteins that are involved in early brain development, but are no longer present in the adult brain may provide a new strategy to prevent progressive degeneration of the nerve cells in the brain that cause Parkinson's disease," said Pedro Lowenstein, M.D., Ph.D., Director of the Gene Therapeutics Research Institute at Cedars-Sinai Medical Center, and a Professor of Medicine and Pharmacology at UCLA.

Parkinson's disease occurs when the nerve cells in the part of the brain known as the "substantia nigra" that produce a chemical called dopamine begin to malfunction and progressively die. Dopamine acts as a chemical messenger to send signals to a part of the brain called the basal ganglia that controls movement and coordination. But as more of these cells die, less and less dopamine is produced, causing Parkinson's disease.

To treat the symptoms of Parkinson's disease, doctors rely on various types of drugs that work by helping to replenish dopamine. A drug called L-DOPA (
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Contact: Kelli Hanley
kelli.hanley@cshs.org
310-423-3674
Cedars-Sinai Medical Center
8-Sep-2004


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