Article
TALEN-Induced Double-Strand Break Repair of CTG Trinucleotide Repeats.
Cell reports - 20 Feb 2018
Mosbach Valentine, Poggi Lucie, Viterbo David, Charpentier Marine, Richard Guy-Franck
Abstract excerpt
Trinucleotide repeat expansions involving CTG/CAG triplets are responsible for several neurodegenerative disorders, including myotonic dystrophy and Huntington's disease. Because expansions trigger the disease, contracting repeat length could be a possible approach to gene therapy for these disorders. Here, we show that a TALEN-induced double-strand break was very efficient at contracting expanded CTG repeats in...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
