Gene Therapy And Neuroprotection

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Our laboratory now produces adeno-associated viral (AAV) vectors for gene delivery. We have obtained the reagents needed to package 7 different serotypes of AAV vectors thereby offering more selection in the type of cells/tissues that receive our transgene of interest. One use of these vectors is to delivery genes to neurons in culture. We are currently characterizing the tropism and toxicity of the various serotypes in primary neuronal cultures. These studies will establish the appropriate serotype and MOI to use to transduce our genes of interest. Using these infection parameters we can deliver various genes to in vitro models of methamphetamine-toxicity, excitotoxicity, hypoxia and Parkinson?s disease to characterize the signal transduction pathways involved in the protective and regenerative properties of bone morphogenetic proteins (BMPs). Our second approach uses AAV vectors for in vivo delivery to the brain in models of stroke, Parkinson?s disease and methamphetamine-toxicity. Specifically, we evaluate the protective and regenerative properties of BMP7 and its signaling molecules. Currently, we are testing the role of AAVBMP7 and AAVSMADs in models of Parkinson?s and methamphetamine-toxicity.