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New gene therapy technique shows promise in stem cell model of retinitis pigmentosa

June 11, 2020

Although the 2017 FDA approval of Spark Therapeutics’ gene therapy Luxturna was considered a triumph for people with inherited eye disease, the product can only be used in patients with mutations in the RPE65 gene. Now, scientists in the United Kingdom say they’ve developed a gene therapy technique that could help a larger population of patients with retinitis pigmentosa.

Scientists at Trinity College Dublin and University College London zeroed in on the RP2 gene, which can become mutated to cause a number of forms of retinitis pigmentosa.

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