Friedreich Ataxia and close related scientific news. Topics related to rare diseases.
Thursday, April 11, 2024
Expression and processing of mature human frataxin after gene therapy in mice
Rojsajjakul, T., Selvan, N., De, B. et al. Expression and processing of mature human frataxin after gene therapy in mice. Sci Rep 14, 8391 (2024). doi:10.1038/s41598-024-59060-0
AAVrh.10hFXN induced mature hFXN expression in mouse heart and liver at levels that approximated endogenous mFXN levels. These results suggest that AAVrh.10hFXN can likely induce expression of therapeutic levels of mature hFXN in mice.