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HomeminewsScientists Collaborate for Usher Syndrome

Scientists Collaborate for Usher Syndrome

A prestigious Medical Research Future Fund (MRFF) grant is enabling leading experts in eye and ear clinical care and research, from three universities and two medical institutes, to coordinate investigations into Usher syndrome in Western Australia.

As part of the project, scientists will create retinal and inner ear organoids, which replicate the eye and ear tissue of a person with Usher syndrome.

The organoids will be used to test a range of drug and gene therapy treatments for Usher syndrome currently being developed by leading research groups at The University of Western Australia (UWA), Curtin University and Murdoch University. This pre-clinical testing is critical to identify safe and effective treatments that are suitable for progression into human clinical trials.

“We are producing cells that are affected by Usher syndrome in order to understand how the disease works and test various strategies for correction,” said Dr Elaine Wong, Principal Researcher at Ear Science Institute Australia. “The organoids can take up to a year to generate from skin cells harvested from patients. The research will be expedited with the arrival of a stem cell robot at the Lions Eye Institute in 2023.”

The organoids will form a common platform for the evaluation of a diverse range of therapeutic technologies being developed in Western Australia, including the latest developments in nanoparticle gene delivery systems from Curtin University, antisense oligomer drug treatments from Murdoch University, as well as gene replacement and gene editing therapies being developed at UWA and the Lions Eye Institute.