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CNS
Application Projects
Project: Central
Nervous System Trauma
Paul Reier, Ph.D.
Description in development.
Project: Parkinson's
Ron Mandel, Ph.D.
Active research is being done in the application of gene transfer to
study animal models of neurodegenerative disorders including Parkinson's
disease, Huntington's disease, Alzheimer's disease, brain tumors, and
ischemia.
Two different strategies will be investigated
to alleviate behavioral deficits in rat models of Parkinson's disease
(PD). Both direct intrastriatal transmitter replacement (L-dopa delivery)
and neurotrophic support strategies (GDNF delivery) in the unilateral
6-OHDA lesion model of PD using recombinant adeno-associated viral vectors
(rAAV) are being pusrued. There is a plan to begin analogous studies
in primates to obtain the data necessary to support a Phase I clinical
trial using rAAV in PD. This field has been greatly engergized lately
by very positive findings reported from Jeffrey Kordower's laboratory
using a recombinant lentivirus to deliver GDNF to the rhesus monkey striatum.
Very important studies are underway to develop regulated vectors to express
GDNF.
The laboratory will begin experiments using rAAV to
transfer ribozymes to the striatum of transgenic mice expressing a mutant form
of huntingtin to determine if knocking down gene expression will block Huntington's
disease-like pathology. Other potential projects involving gene transfer in Huntington's
disease models concern the trophic factors brain derived neurotrophic factor
(BDNF) and ciliary neurotrophic factor (CNTF).
Project:
Alzheimer's
Ed Meyer, Ph.D.
Description in development.
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