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Increased Frataxin Expression Induced in Friedreich Ataxia Cells by Platinum TALE-VP64s or Platinum TALE-SunTag

Frataxin gene (FXN) expression is reduced in Friedreich’s ataxia patients due to an increase in the number of GAA trinucleotides in intron 1. The frataxin protein, encoded by that gene, plays an important role in mitochondria’s iron metabolism. Platinum TALE (plTALE) proteins targeting the regulator...

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Detalles Bibliográficos
Autores principales: Cherif, Khadija, Gérard, Catherine, Rousseau, Joël, Ouellet, Dominique L., Chapdelaine, Pierre, Tremblay, Jacques P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6019861/
https://www.ncbi.nlm.nih.gov/pubmed/30195758
http://dx.doi.org/10.1016/j.omtn.2018.04.009