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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Bibliographic Details
Published in:Mol Ther Nucleic Acids
Main Authors: Cherif, Khadija, Gérard, Catherine, Rousseau, Joël, Ouellet, Dominique L., Chapdelaine, Pierre, Tremblay, Jacques P.
Format: Artigo
Language:Inglês
Published: American Society of Gene & Cell Therapy 2018
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Article
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC6019861/
https://ncbi.nlm.nih.gov/pubmed/30195758
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2018.04.009
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