Sustained Effects of Nonallele-Specific Huntingtin Silencing

Autor: Drouet, Valerie, Perrin, Valerie, Hassig, Raymonde, Dufour, Noelle, Auregan, Gwennaelle, Alves, Sandro, Bonvento, Gilles, Brouillet, Emmanuel, Luthi-Carter, Ruth, Hantraye, Philippe, Deglon, Nicole
Předmět:
Popis: Objective: Huntington's disease (HD) is a fatal autosomal dominant neurodegenerative disorder caused by a polyglutamine expansion in the huntingtin (htt) protein. No cure is available to date to alleviate neurodegeneration. Recent studies have demonstrated that RNA interference represents a promising approach for the treatment of autosomal dominant disorders. But whether an allele-specific silencing of mutant htt or a nonallele-specific silencing should be considered has not been addressed.
Databáze: OpenAIRE