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).
Goureau et al. (Mon,) studied this question.