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Using patient-specific stem cells to model optic neuropathies

Research Unit: Cellular Reprogramming
Primary supervisor: Dr Raymond Wong
Tel: +613 99298054 / Email : wongcb@unimelb.edu.au

Optic neuropathies represent a group of ocular diseases characterised by loss of retinal ganglion cells. The extreme difficulty in obtaining ocular tissue from living individuals, as well as the lack of good cellular models, represent major barriers to studying and treating optic neuropathies. Recent advances now allow us to reprogram patient’s somatic cells into induced pluripotent stem (iPS) cells, which can give rise to all cell types in the body and proliferate indefinitely in vitro. This project will direct patient-specific iPS cells into retinal ganglion cells to create an in vitro model for studying optic neuropathies. In particular, the student will undertake studies with other team members to investigate the molecular mechanisms underlying disease progression of optic neuropathy, development of reprogramming and differentiation technologies to generate patient-specific retinal ganglion cells, and contribute to the development of ex vivo model to test gene therapy and cell therapy, as well as drug screening to identify bioactive compounds that can rescue the disease in iPS-derived retinal ganglion cells from patients to develop novel treatments for optic neuropathies.

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