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Article

Expression and processing of mature human frataxin after gene therapy in mice

Friedreich’s ataxia is a degenerative and progressive multisystem disorder caused by mutations in the highly conserved frataxin (FXN) gene that results in FXN protein deficiency and mitochondrial dysfunction. ...

Teerapat Rojsajjakul, Nithya Selvan, Bishnu De, Jonathan B. Rosenberg… in Scientific Reports (2024)