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Development of regenerative therapy for photoreceptor losses using cell reprogramming technology

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

Photoreceptors are light-sensing cells that form the basis of our vision by converting light into electrical signals that can be decoded by the brain.  the loss of photoreceptors is a key hallmark of many blinding diseases, such as retinitis pigmentosa, age-related macular degeneration, and diabetic retinopathy.  These diseases affect millions of patients and cause a significant socis-economic burden on our healthcare system. Currently, there are no effective means to cure blindness once photoreceptors are lost.  We must therefore find a new approach to help restore vision to these patients.  Regenerative therapy to replace photoreceptors has the very real prospect of helping patients to restore vision.

Cell reprogramming could be the key to this critical issue.  This innovative technology relies on converting one cell type into another by rewriting the transcriptome to alter the cell’s identity.  One of the most famous examples is the Nobel prize-winning discovery of induced pluripotent stem (iPS) cells, in which the altered expression of four transcription factors converted adult fibroblasts into stem cells.  Beyond iPS cells, direct reprogramming is now possible by converting one somatic cell type directly to another, such as fibroblasts to neurons, without passing through an intermediate stem cell state.  this project aims to develop cell reprogramming technology to generate new photoreceptors, providing novel regenerative therapy approach to treat photoreceptor loss.  Techniques involved in this project include cell reprogramming, CRISPR/Cas9, transcriptomic analysis, molecular cloning, fluorescent micrscopy and virus generation.

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