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  • Journal Club

Stargardt’s disease: groundbreaking gene therapy study gives hope

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  • 4 minute read

In Stargardt disease, loss-of-function mutations in the ABCA4 gene lead to macular degeneration and blindness. A team led by researchers from the University of Basel has developed a promising new therapeutic approach. In their study, they were able to demonstrate precise gene correction using “base editing”. The results were published in the journal Nature Medicine.

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Autoren
  • Mirjam Peter, M.Sc.
Publikation
  • HAUSARZT PRAXIS
Related Topics
  • Blindness
  • macular degeneration
  • Stargardt's disease
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