Gene therapy research gives hope on World Sight Day

Sisters Nicole and Kate Barrett standing on the beach with Kate's guide dog Misty.

Nicole and Kate Barrett with guide dog Misty.

The potential of gene therapy to prevent blindness and restore sight for eye diseases that have long been considered untreatable is the focus on World Sight Day (8 October 2020) at the Centre for Eye Research Australia.

“Gene and cell therapy are two emerging technologies offering new hope to patients with diseases where no treatments have been available in the past,” says CERA Managing Director Professor Keith Martin.

“This includes people with inherited retinal diseases like retinitis pigmentosa or Stargardt’s disease, or those who have experienced irreversible vision loss due to optic nerve diseases like glaucoma.”

On World Sight Day, 8 October 2020, CERA is conducting its inaugural Hope in Sight Giving Day to raise awareness of this pioneering research and to raise funds for these exciting new therapies to be developed and trialled in Melbourne.

About 16,500 Australians have inherited retinal diseases – caused by genetic faults that lead to loss of retinal cells causing vision loss and blindness. These diseases are the leading cause of blindness in working-age adults and there are currently no treatments or cures.

However, the emergence of gene and cell therapy is seeing new treatments being trialled internationally – and the chance of stopping vision loss and restoring sight is now a realistic possibility.

Sisters Kate and Nicole Barrett are the faces of our Hope in Sight Giving Day campaign. Kate, 35, was first diagnosed with retinitis pigmentosa at six and now lives with a tiny amount of tunnel vision.

Nicole, 33, an occupational therapist, was diagnosed at 17. Her vision is currently stable, and she lives from day to day hoping her vision will not deteriorate further.

“The new gene therapy research gives me hope that in my lifetime there will be a treatment for retinitis pigmentosa,” says Kate, who lives on a farm in Gippsland with her husband and 10-year-old twin daughters.

“It gives everybody hope to know that scientists are looking into all avenues for a cure. My wish is for my sight can stay where it is so I can see the milestones in my daughters’ lives.”

Research underway at CERA to tackle irreversible blindness includes:

  • Development of a gene therapy to tackle a rare form of retinitis pigmentosa
  • Gene therapy to repair optic nerve damage that causes blindness in glaucoma
  • Stem cell therapies to regenerate light sensing cells in the eye to restore sight
  • Tissue engineered corneas for transplant
  • A study to understand how IRDs progress and identify people suitable for clinical trials

Gene therapy researcher Dr Tom Edwards says it is exciting to offer patients hope that there are treatments on the horizon.

“Gene therapy research offers the best prospect of finding cures for inherited eye diseases that cause blindness,” he says.

Every dollar donated to CERA’s research on World Sight Day will be tripled by generous matched on donations from the quotes from National Stem Cell Foundation of Australia and the Centre for Eye Research Australia Foundation.

“We’re particularly excited by the potential for Dr Tom Edward’s gene therapy approach to be applied to many different inherited eye diseases, not just one,” said National Stem Cell Foundation of Australia Chairman Dr Graeme Blackman AO.

“We encourage people to join us in making a donation to support the research of Tom and his colleagues.”

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