Patient-derived iPSC models of Friedreich ataxia: a new frontier for understanding disease mechanisms and therapeutic application

Friedreich ataxia (FRDA) is a rare genetic multisystem disorder caused by a pathological GAA trinucleotide repeat expansion in the FXN gene. The numerous drawbacks of historical cellular and rodent models of FRDA have caused difficulty in performing effective mechanistic and translational studies to...

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Detalles Bibliográficos
Autores principales: Maheshwari, Saumya, Vilema-Enríquez, Gabriela, Wade-Martins, Richard
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10510273/
https://www.ncbi.nlm.nih.gov/pubmed/37726850
http://dx.doi.org/10.1186/s40035-023-00376-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10510273/
https://www.ncbi.nlm.nih.gov/pubmed/37726850
http://dx.doi.org/10.1186/s40035-023-00376-8

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