Sydney, New South Wales, Australia
There are currently many incurable inherited diseases, and stem cell approaches offer significant promise towards therapies for these conditions. My research has always been focused in two complementary fields of research: stem cells and regenerative medicine. Throughout my career I have been involved in a number of stem cell projects including disease modelling and the development of gene and cell therapies. The Stem Cell Medicine Unit focuses on three main areas: (1) disease modelling of inherited retinal, auditory and brain degenerations (2) understanding the development of the human retina using bioengineered complex retinal organoids (3) developing novel cell therapies to treat inherited retinal diseases due to the loss of photoreceptor cells We use 3D protocols to differentiate organoids from human pluripotent stem cell (hPSC) to model disease in the dish. We aim to understand the pathophysiology of retinal and otic inherited degenerations as well as develop new gene therapy treatments for Usher syndrome and Stargardt’s maculopathies. We are specifically interested in understanding the development of the human macula to generate this human eye specific region in retinal organoids in vitro. We use novel paradigms, such as electrical stimulation to develop better models in vitro and to promote cellular integration following cell transplantation.