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Stephen Tsang, Associate Professor, Ophthalmology, Pathology & Cell Biology, Columbia University, “Patient-specific stem cells as a platform for precision medicine”
November 6, 2018 @ 12:00 pm - 1:00 pm
Topic: Patient-specific stem cells as a platform for precision medicine
Abstract: In the current era of personalized medicine, a large number of genetic variants have been discovered in patients with various diseases using next-generation sequencing techniques. Traditionally, to prove that genetic variants cause diseases, we have had to rely on animal models. However, substantial differences exist between mice and humans, including but not limited to drastic differences in lifespans. For instance, dopaminergic neuron projections are shorter in relation to overall body length in mice than in humans; thus, alpha synuclein Snca knockout mice do not develop Parkinson disease. Instead, as new reprogramming technologies have developed, it has become more feasible to generate patient-specific stem cell lines to validate sequence variants, elucidate pathophysiology, and perform targeted drug screening.
Other than cancer, blindness is the most feared of condition amongst Americans. The eye is an ideal testing ground for stem cell therapies because of its relative immune privilege and its ready accessibility for monitoring and imaging purposes. In the eye, various assays can be used to non-invasively quantify transplanted tissue at multiple time points. Furthermore, insight gained from the study of retinal disorders is applicable to other diseases and organ systems, thereby promoting the development of novel stem cell- and gene-based cures for a broad array of previously incurable maladies.