Fine-tuning FAM161A gene augmentation therapy to restore retinal function
Yvan Arsenijévic, Ning Chang, Olivier Mercey, Younes El Fersioui, Hanna Koskiniemi-Kuendig, Caroline Joubert, Alexis‐Pierre Bemelmans, Carlo Rivolta, Eyal Banin, Dror Sharon, Paul Guichard, Virginie Hamel, Corinne Kostic
This study fine-tunes FAM161A gene therapy in mice to restore retinal function by optimizing promoter activity, dosage, and isoform delivery.
EMBO Molecular Medicine · Mar 19, 2024Peer-reviewedNeuroscience