Cell Genesys Scientists Demonstrate Prevention of Neurodegeneration In
Parkinson's Disease Model
FOSTER CITY, Calif., Dec. 9 /PRNewswire/ -- Cell Genesys, Inc.
(Nasdaq: CEGE) today announced that a team of scientists, led by Ronald J.
Mandel, Ph.D., successfully prevented neurodegeneration in an animal model for
Parkinson's disease utilizing adeno-associated viral (AAV) gene therapy. AAV
vectors were used to deliver the gene for glial cell line-derived neurotrophic
factor (GDNF) to the region of the brain affected in Parkinson's disease
resulting in stable expression of the GDNF gene measured out to ten weeks, the
duration of the study. The study was published in the December 9, 1997 issue
of the journal, Proceedings of the National Academy of Sciences.
"The long-term expression we are observing using AAV-based gene delivery
is very exciting because just a single injection of this gene therapy may
achieve an ongoing therapeutic effect in various disease targets such as
Parkinson's disease," stated Mitchell H. Finer, Ph.D., vice president,
research at Cell Genesys. "In this and other recently published studies, AAV
vectors have been used to successfully deliver genes to the liver, neurons and
muscle tissue with prolonged gene expression demonstrated for each tissue
type, creating potential product opportunities in multiple diseases and
genetic disorders."
In this published work, Cell Genesys scientists utilized AAV vectors to
deliver the GDNF gene to a specific region of the brain of the animals
affected with Parkinson's disease. The site specific delivery of GDNF was
shown to prevent the degeneration of the nerve cells responsible for the
production of the neurotransmitter, dopamine, the absence of which results in
the symptoms of Parkinson's disease. Through a single injection of AAV gene
therapy, stable expression of the GDNF protein was measured for the duration
of the study, effectively protecting the dopamine neurons from degeneration.
Parkinson's disease is a chronic degenerative neurologic condition affecting
movement and speech. Cell Genesys is currently seeking a corporate partner
for the further development of its Parkinson's disease gene therapy program.
Utilizing AAV vectors, company scientists have observed long-term
expression in various cell types including liver cells, muscle cells and
neurons which may allow for a broad variety of potential disease targets
including hemophilia, anemia, cardiovascular and central nervous system
disorders. The company recently received a notice of broad patent claims
covering gene therapy products that utilize AAV vectors to deliver genes as
therapeutic agents.
AAV vectors are one of several gene delivery systems being developed by
Cell Genesys. The company is using retroviral vectors in its human clinical
trials of the company's T cell gene therapy for AIDS and cancer. Adenoviral
vectors are being employed in the company's GVAX(TM) cancer vaccine clinical
trials. Lentiviral vector technology is also being developed by the company.
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SOURCE Cell Genesys, Inc.
