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, Daria V. Mamaeva, Marie O. Péquignot, Nejla Erkilic, Carla Sanjurjo-Soriano, Hassan Boukhaddaoui, Florence Bernex, Béatrice Bocquet, Jérôme Vialaret, Yvan Arsenijévic, T. Michael Redmond, Christophe Hirtz, Isabelle Meunier, , 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.
Molecular Therapy · Oct 9, 2024Peer-reviewedMolecular Biology