In our lab, we develop and test stem cell and gene therapeutic cures for Parkinson’s disease using the best animal model of the disease.
Through the differentiation of pluripotent stem cells into neurons we hope to replenish the dopamine loss associated with the degenerative effects of PD. After transplanting the cells into our animal model we are able to perform a variety of tests to identify if they are effective in restoring dopamine levels. Achieving that goal could reverse the symptoms associated with Parkinson’s.
Previously, we used fetal brain tissue, but now we use stem cells both alone and in conjunction with gene therapies. We use an adeno-associated viral vector (AAV) to express a growth factor, GDNF, which increases neural production of dopamine and the survival rate of our cells. It also may improve directional outgrowth of synaptic processes from the cells.