A knockdown-and-replace gene therapy from Penn researchers shows promise in rare eye disease
It’s been a year of great strides in gene therapy, with Spark’s Luxturna earning the first US regulatory approval in the field for inherited blindness. Now, researchers at University of Pennsylvania might be able to add to that success with a new gene therapy that’s shown promise tackling a group of eye problems called retinitis pigmentosa.
This rare condition changes how the retina responds to light, making it hard for people to see — especially affecting night vision and peripheral vision. And it gets worse over time, sometimes leading to near-blindness. Scientists have identified more than 150 mutations of the light-sensing molecule rhodopsin, which all lead to the disease. However, those researchers at Penn intend to develop a treatment that works regardless of the mutation. They’ve published their early-stage findings in this week’s Proceedings of the National Academy of Sciences.
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