Neurodegenerative Diseases, Stem Cells, Parkinson's Disease

Research in my laboratory is aimed at understanding how neurons differentiate into dopamine neurons during development of the brain and how that information may be useful for the treatment of neurodegenerative diseases such as Parkinson's. A major goal of our studies has been defining the key fate determinant genes and lineage stages in the development of human dopamine neurons. Our hope is that an understanding of those mechanisms that first direct expression of neurotransmitter genes during differentiation will provide a molecular blueprint that can be used to intentionally target the differentiation of cells, such as human embryonic stem or precursor cells, toward that phenotype. Using an approach that combines cell culture and genetic engineering, our aim is to induce dopaminergic traits in human stem/progenitor cells and devise ways to amplify and purify prospective human dopamine neurons for study after transplantation into rat and monkey models of Parkinson's disease.

In our laboratory, we use a multidisciplinary approach, employing tissue culture (primary and cell lines), molecular (qPCR, microarray, gene cloning, transfection, transduction), anatomical (immunocytochemistry, confocal) biochemical (HPLC), surgical (stereotaxic brain surgery, arterial occlusion), imaging (PET, spect) and behavioral (sensory and motor skills tests).

The overall goal of our studies is take what we have learned about the differentiation of dopamine neurons from human stem cells and translate that into a cell replacement treatment for Parkinson's disease.