Molecular therapy : the journal of the American Society of Gene Therapy
Development of an AAV-based gene therapy for the ocular phenotype of Friedreich's ataxia.
Heyu Tang, Siddhant Gupte, Emily Xu, Kaitlyn R Calabro, Hannah Friend, Sean M Crosson, Diego Fajardo, Zachary Kostamo, Hangning Zhang, James J Peterson, Fangyu Lin, Zbynek Kozmik, Cathleen M Lutz, Sanford L Boye, Shannon E Boye
Published: 202510.1016/j.ymthe.2025.10.048
Abstract
Friedreich's ataxia (FA) is a leading form of hereditary ataxia caused by autosomal recessive mutations in frataxin (FXN). GAA triplet repeat expansions lead to lower levels of FXN expression, abnormal influx of iron into mitochondria, and damage to…
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