Stem cell research & therapy
Human pluripotent stem cell models of Friedreich's ataxia: innovations, considerations, and future perspectives.
Ha Thi Nguyen, Marek Napierala, Jill S Napierala
Published: 202610.1186/s13287-025-04861-x
Abstract
Friedreich's ataxia (FRDA) is an inherited, autosomal recessive, multisystem disorder that primarily manifests in children and affects the nervous system and the heart. FRDA is caused by an expansion of GAA repeats in the first intron of the frataxin…
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