Gene therapy for retinitis pigmentosa could be on the horizon

  • University of Leeds
  • 12 Oct 2018

  • curated by Pavankumar Kamat
  • UK Medical News
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New in-vitro research, published in the journal Nature Communications, has shown that gene editing could be possibly used to fix degenerated retinal cells. This could open new avenues in developing a treatment for retinitis pigmentosa, the most common form of inherited blindness affecting ~23,000 individuals in the United Kingdom and up to 2.5 million globally.

Researchers from the Universities of Leeds and Newcastle creating an invitro retina using stem cells derived from skin samples donated by patients at Leeds Teaching Hospitals Trust. They found that errors in "proofreading" the genetic code of retinal cells result in mutations responsible for retinitis pigmentosa. They hope that this new understanding of the disease pathogenesis could pave the way for developing a gene therapy for the condition.

Speaking about their discovery, co-author Professor Colin Johnson, said: "We've been puzzled by the genetics behind these unique forms of inherited blindness for over 20 years. We are very hopeful that this will lead to clinical trials for new treatments within five years."

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