Melbourne researchers aim to use stem cells to reverse blindness
STEM cells are emerging as a new approach to reversing blindness, with Melbourne researchers aiming to reprogram diseased eye cells into healthy ones.
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STEM cells are emerging as a new approach to reversing blindness, with Melbourne researchers aiming to reprogram diseased eye cells into healthy ones that can be transplanted back into the patient.
The Centre for Eye Research Australia team has jumped the first hurdle in this ambitious plan, taking skin cells from patients with eye disease and reprogramming them into specialist eye cells.
This source of diseased optic nerve cells can be used to test ways of correcting disease-causing genetic defects, as well as trialling new drugs and combinations that can’t be tested in patients.
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The team is also developing a proof-of-concept way to replace damaged mitochondria — the energy powerhouses of the cell that work less efficiently in many eye diseases — so the problem can be reversed in the Petri dish.
Head of CERA’s Cellular Reprogramming Unit, Dr Raymond Wong, said new treatments and drugs needed to be tested in preclinical models before humans but until now, there had not been a source of diseased optic nerve cells available to researchers to test treatments in the laboratory.
Their 45-day technique to grow these retinal ganglion cells is published in the Nature journal Scientific Reports.
As well as reprogramming the cells, Dr Wong said these optic nerve cells would first be used to test drug treatments for the inherited and incurable blindness, Leber hereditary optic neuropathy.
The disease, which typically causes teenage boys to go blind in weeks, is caused by a genetic mutation in the cell’s mitochondria, which causes the death of cells in the optic nerve.
“The concept of disease modelling is very simple. We wanted to get optic nerve cells, the cells affected by the disease, but we can’t get patients to donate their optic nerve cells,” Dr Wong said.
“We can take skin or hair cells from the patient, and use a technique called reprogramming to turn them into stem cells. These stem cells can become any cell in the body, and we turn them into optic nerve cells in the dish.
“If we can develop new drugs to correct the cells, that could be a novel treatment, or if we can correct the genetic defects then we could potentially transplant them back to the patient.”
Dr Wong said if the work proved successful in LHON, the technology could apply to other optic nerve diseases.
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