Quantification of human mature frataxin protein expression in nonhuman primate hearts after gene therapy

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Deficiency in human mature frataxin (hFXN-M) protein is responsible for the devastating neurodegenerative and cardiodegenerative disease of Friedreich’s ataxia (FRDA). It results primarily through epigenetic silencing of the FXN gene by GAA triplet repeats on intron 1 of both alleles. GAA repeat len...

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Detalles Bibliográficos
Autores principales: Rojsajjakul, Teerapat, Hordeaux, Juliette J., Choudhury, Gourav R., Hinderer, Christian J., Mesaros, Clementina, Wilson, James M., Blair, Ian A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10611776/
https://www.ncbi.nlm.nih.gov/pubmed/37891254
http://dx.doi.org/10.1038/s42003-023-05472-z
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