Could this be what Tom Brokaw was trying to tell us???
Company Press Release
SOURCE: Cell Genesys, Inc.
Cell Genesys Reports Encouraging Results in Preclinical Studies of
Parkinson's Disease Gene Therapy
Single Gene Therapy Treatment Eliminates the Need for Daily L-dopa
Treatment
FOSTER CITY, Calif and SEATTLE, Jan. 28 /PRNewswire/ -- Cell Genesys,
Inc. (Nasdaq: CEGE - news) and the University of Washington today
published data demonstrating that after a single gene therapy injection,
genetically modified mice exhibiting certain symptoms of Parkinson's
disease could survive without daily L-dopa treatments for at least one
year, the duration of the study. Without L-dopa treatment, these mice
die of malnutrition by three weeks of age. An adeno-associated viral
(AAV) gene delivery system was used to deliver the genes required for
the production of L-dopa to specific regions of the brain where L-dopa
production could be detected throughout the observation period. L-dopa
is a commonly prescribed drug which is converted to dopamine, the
neurotransmitter chemical missing in these mice and in patients with
Parkinson's disease. This work was published in the journal, Neuron, by
a team of scientists led by Mark Szczypka, Ph.D. and Richard Palmiter,
Ph.D. at the University of Washington and Ronald J. Mandel, Ph.D. and
Richard O. Snyder, Ph.D. of Cell Genesys.
``A single gene therapy treatment for Parkinson's disease would be a
significant improvement over the currently available treatment for this
disease,'' stated Mitchell H. Finer, Ph.D., vice president, research at
Cell Genesys. ``These
studies are among the first observation that gene therapy with AAV
vectors can be used to correct a genetic defect in specific regions of
the brain.''
``The most striking finding in our experiments was the elimination of
the need for daily L-dopa treatment in the mice receiving the gene
therapy. This represents a remarkable rescue of an otherwise lethal
mutation in these genetically
modified mice,'' stated Dr. Richard Palmiter, Investigator of the Howard
Hughes Medical Institute and Professor of Biochemistry at the University
of Washington. ``In the published work, the motivation of the mice to
eat and drink was restored following the single administration of gene
therapy.''
In the published experiments, mice dependent upon daily injections of
L-dopa for their survival, were injected with AAV vectors carrying the
tyrosine hydroxylase and GTP cyclohydrolase I genes directly to the
striatum, one of the principal regions of the brain affected in
Parkinson's disease. L-dopa was then produced at therapeutic levels by
the genetically modified brain cells for the duration of the study
making daily treatment with exogenous L-dopa unnecessary. These data
further support previous findings that both of these two genes are
required for L-dopa
synthesis in the brain and suggest a potential treatment strategy for
patients with Parkinson's disease.
The mouse model used in this study was created in Dr. Palmiter's
laboratory using gene-targeting techniques. The genetically modified
mice are unable to make tyrosine hydroxylase, a critical enzyme for
dopamine synthesis in the brain. These mice develop severe movement
dysfunction that resembles that of Parkinson's disease patients. The
mice also have behavioral defects and will not eat and drink enough to
survive without L-dopa therapy.
--
Judith Richards, London, Ontario, Canada
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