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

ABSTRACT: 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 regulatory region of the FXN gene, fused with a transcriptional activator (TA) such as VP64 or P300, were used to increase the expression of that gene. Many effectors, plTALE_VP64, plTALE_p300, and plTALE_SunTag, targeting 14 sequences of the FXN gene promoter or intron 1 were produced. This permitted selection of 3 plTALE_VP64s and 2 plTALE_SunTag that increased FXN gene expression by up to 19-fold in different Friedreich ataxia (FRDA) primary fibroblast

SUBMITTER: Cherif K 

PROVIDER: S-EPMC6019861 | biostudies-literature | 2018 Sep

REPOSITORIES: biostudies-literature