Article
Self-duplexing CUG repeats selectively inhibit mutant huntingtin expression.
Nucleic acids research - 1 Dec 2013
Fiszer Agnieszka, Olejniczak Marta, Galka-Marciniak Paulina, Mykowska Agnieszka, Krzyzosiak Wlodzimierz J
Abstract excerpt
Huntington's disease (HD) is a neurodegenerative genetic disorder caused by the expansion of the CAG repeat in the translated sequence of the HTT gene. This expansion generates a mutant huntingtin protein that contains an abnormally elongated polyglutamine tract, which, together with mutant transcript, causes cellular dysfunction. Currently, there is no curative treatment available to patients suffering from HD;...
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