ELP1 Gene Augmentation Restores Visual Function in a Mouse Model of Familial Dysautonomia
Hui‐Chen Cheng, Swanand Koli, Kate J. Lewis, Sara Ketchale, Yasaman Anvarinia, Morgan P. Erdoes, Yating Liu, Caitlin Elizabeth Keiper, Reynette Estelien, Matthew Chagnon, Luk H. Vandenberghe, Dadi Gao, Frances B. Lefcort, Elisabetta Morini, Susan A. Slaugenhaupt, Anil K. Chekuri
This study shows that gene supplementation with ELP1 in a mouse model of familial dysautonomia rescues retinal structure and function, suggesting it as a potential therapy for FD-associated optic neuropathy.
Molecular Therapy · Apr 25, 2026Peer-reviewedGenetics