In vivo overexpression of frataxin causes toxicity mediated by iron-sulfur cluster deficiency

Friedreich's ataxia is a rare disorder resulting from deficiency of frataxin, a mitochondrial protein implicated in the synthesis of iron-sulfur clusters. Preclinical studies in mice have shown that gene therapy is a promising approach to treat individuals with Friedreich's ataxia. However...

Descripción completa

Detalles Bibliográficos
Autores principales: Huichalaf, Claudia, Perfitt, Tyler L., Kuperman, Anna, Gooch, Renea, Kovi, Ramesh C., Brenneman, Karrie A., Chen, Xian, Hirenallur-Shanthappa, Dinesh, Ma, Tiffany, Assaf, Basel T., Pardo, Ingrid, Franks, Tania, Monarski, Laura, Cheng, Ting-Wen, Le, Kevin, Su, Chunyan, Somanathan, Suryanarayan, Whiteley, Laurence O., Bulawa, Christine, Pregel, Marko J., Martelli, Alain
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8866050/
https://www.ncbi.nlm.nih.gov/pubmed/35252470
http://dx.doi.org/10.1016/j.omtm.2022.02.002

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8866050/
https://www.ncbi.nlm.nih.gov/pubmed/35252470
http://dx.doi.org/10.1016/j.omtm.2022.02.002

Ejemplares similares