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Researchers develop gene therapy for rare ciliopathy

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NIH researchers develop gene therapy for rare ciliopathy

Treating patient-derived retinal organoids with AAV-NPHP5 restores rhodopsin localization in photoreceptor outer segments. Top: Retinoids stained for DNA (blue), NPHP5 (red), and rhodopsin (green). Below: Close-up view of organoid photoreceptor layer stained green for rhodopsin. Adapted from Kruczek et al, 2022. Credit: Anand Swaroop, Ph.D. and Kamil Kruczek, Ph.D.,…

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