A Potential New Therapeutic Approach for Friedreich Ataxia: Induction of Frataxin Expression With TALE Proteins

TALEs targeting a promoter sequence and fused with a transcription activation domain (TAD) may be used to specifically induce the expression of a gene as a potential treatment for haploinsufficiency. This potential therapeutic approach was applied to increase the expression of frataxin in fibroblast...

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Hlavní autoři: Chapdelaine, Pierre, Coulombe, Zoé, Chikh, Amina, Gérard, Catherine, Tremblay, Jacques P
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group 2013
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Original Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC4028015/
https://ncbi.nlm.nih.gov/pubmed/24002729
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtna.2013.41
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