[MOL] pam: parkinson article [03344] Medicine On Line

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[MOL] pam: parkinson article


   A single gene therapy injection given to mice with Parkinson's
disease prevented the need for therapy with the drug L-Dopa for a full
year, scientists said yesterday. Normally, such mice wouldn't survive
longer than three weeks without the drug.
    The study, done at the University of Washington, offers promise for
a half-million Americans with Parkinson's, a movement disorder
characterized by muscle rigidity, tremors and changes in behavior. It
was published today in the journal Neuron.
     It's long been known that people with Parkinson's lose virtually
all of the dopamine-containing cells in an area of the brain called the
substantia nigra. L-Dopa helps replace dopamine, but the therapy is
effective only as long as dopamine-containing cells are present, and
symptoms don't usually begin until more than 80 percent of these cellshave
    The challenge has been to keep dopamine-containing cells alive
longer so doctors can better manage the disabling symptoms, which turn
on and off throughout the day.
    The study used mice that were genetically engineered without a
critical enzyme, tyrosine hydroxylase, that's needed to produce
dopamine, prompting the classic signs of Parkinson's. The researchers
injected genes that produced two enzymes critical for dopamineproduction.
    Dr. Stephen Sherwin, president of Cell Genesys, the Foster City,
Calif., biotech firm that developed the gene system, said the study
"shows what gene therapy in the brain can do. It creates an on-going
production of a substance with disease-fighting properties."
    "The most striking finding was the elimination of the need for daily
L-dopa treatment," said Richard Palmiter, a biochemist and co-author of
the new study. More animal studies are necessary before the gene therapy
is ready for testing in humans.

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