Demonstrates enhanced photoreceptor protection in retinitis pigmentosa using combined gene therapy in a gene therapy trial.
This study revealed that both proteins encoded by NXNL1 are essential for gene-independent RP treatment and that combining RdCVF and RdCVFL delivery with a single AAV vector yields a more substantial photoreceptor-protective effect. This functional proof of concept supports the ongoing gene therapy phase 1/2 trial Promising ROd-cone DYstrophy Gene therapY (PRODYGY, NCT05748873).
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Goureau et al. (2026) studied this question.
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