Gene therapy
Lipid nanoparticle mediated base editing of the Q344X rhodopsin mutation associated with retinitis pigmentosa.
Victoria A C Palmgren, Miffy Hok Yan Cheng, Yao Zhang, Tiffany Carlaw, Tessa Morin, Jerry Leung, Colin Jd Ross, Pieter R Cullis, Robert S Molday
Published: 202510.1038/s41434-025-00584-z
Abstract
Retinitis pigmentosa (RP) associated with mutations in the rhodopsin gene (RHO) is a significant cause of blindness. Here we report on the application of adenine base editing of the c.1030C>T (p.Q344X) RHO mutation linked to RP. Using a fluorescence…
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