2018 SRF Summer Scholar Profile: Lee Rao

Lee Rao

SRF Summer Scholar, Class of 2018

Scripps Research Institute

Hello! I’m Lee Rao, a rising junior studying Computer Science and Biology at the University of Texas at Austin. At UT Austin, I’m a member of Dr. Claus Wilke’s lab, where I develop algorithms for simulating and analyzing DNA sequences. This summer, I’ll be interning with Dr. Jeanne Loring at The Scripps Research Institute in La Jolla, California. The Loring Lab’s research focuses on pluripotent stem cells and their wide range of applications, such as the conservation of the Northern white rhinoceros or modeling Fragile-X Syndrome. For my project, I’ll be working with Dr. Roy Williams on a bioinformatics analysis of stem cell-derived neurons to assess the potential of cell replacement therapy for Parkinson’s Disease.

Parkinson’s Disease, a neurodegenerative disorder that impacts motor control, affects approximately 7-10 million people worldwide; it is primarily caused by the loss of dopamine-producing neurons in the brain. The ultimate goal of our research is to replace these lost neurons; our protocol extracts tissue cells from Parkinson’s Disease patients, transforms them into pluripotent stem cells (PSCs), and then matures those stem cells into dopamine-producing neurons that can be safely transplanted back into the patient. However, the process of reprogramming, differentiating, and culturing tissue cells could potentially induce or select for deleterious mutations. These mutations could theoretically be oncogenic, which might cause tumors to develop in the patient’s brain even after a successful transplantation. My project assesses the genomic integrity of the cells created from our protocol to determine if they are appropriate for our proposed Parkinson’s Disease therapy. Moreover, the methodologies that we establish for evaluating these cells can be used to assess safety in other applications of cell replacement therapy, such as macular degeneration and type-1 diabetes.

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