Paving the way to cure Usher Syndrome: landmark findings in new paper

What if we could re-program a patient’s own stem cells to become something new – and use them to cure Usher Syndrome, a chronic genetic condition?

Ear Science researchers have been working with Lions Eye Institute on this world-first stem cell research in patients who have a specific mutation that causes loss of both hearing and vision.

Together they have just put out a paper showing some of their findings to the world.

Head of Basic Research, A/Prof Rodney Dilley, says their findings are a positive landmark for the project.

“Our teams have now successfully developed cell lines – cells of a single type that can grow continuously in the lab – from patients with Usher syndrome. This is a huge landmark, because these stem cell lines will help us identify new treatments in the future.”

Usher Syndrome causes progressive loss of hearing, sight and balance – sometimes at the same time. Currently, there is no cure for the genetic condition, but Ear Science Institute Australia and Lions Eye Institute are working to find one.

The findings have been published in Stem Cell Research.

Source: McLenachan S*, Wong EYM*, Leith F, Moon S-Y, Zhang D, Chen S-C, Zhang X, Thompson JA, McLaren T, Lamey T, De Roach JN, Atlas MD, Dilley RJ, Chen FK. Generation of two induced pluripotent stem cell lines from a patient with compound heterozygous mutations in the USH2A gene. Stem Cell Research, 101420.