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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.
Hilal Lashuel, Nathan Alain Denis Riguet, Lorène Aeschbach
Hilal Lashuel, Jonathan Jean-Pierre Ricci, Rajasekhar Kolla, Andreas Reif, Gopinath Pushparathinam