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.
Autoren
- Mirjam Peter, M.Sc.
Publikation
- HAUSARZT PRAXIS
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