Molecular BiologyMolecular TherapyPeer-reviewed
Dual CRALBP isoforms unveiled: iPSC-derived retinal modelling and AAV2/5-RLBP1 gene transfer raise considerations for effective therapy
Krishna Damodar, Grégor Dubois, Laurent Guillou…·Vasiliki Kalatzis
The paper models RLBP1-related retinal diseases using iPSC-derived RPE and validates a gene replacement strategy, identifying a novel CRALBP isoform with functional significance.