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 by epigenetic silencing the FXN gene due to up to 1400 GAA triplet repeats in intron 1 of both alleles of the g...

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Detalles Bibliográficos
Autores principales: Blair, Ian, Rojsajjakul, Teerapat, Hordeaux, Juliette, Chaudhary, Gourav, Hinderer, Christian, Mesaros, Clementina, Wilson, James
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Journal Experts 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10350221/
https://www.ncbi.nlm.nih.gov/pubmed/37461697
http://dx.doi.org/10.21203/rs.3.rs-3121549/v1

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10350221/
https://www.ncbi.nlm.nih.gov/pubmed/37461697
http://dx.doi.org/10.21203/rs.3.rs-3121549/v1

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