Mutant and reversible silencing of endogenous wild type huntingtin gene by siRNA and its application for treatment of Huntington's disease

摘要

Isolated double-stranded short interfering nucleic acid molecules inhibiting the expression of endogenous wild-type and exogenous human mutated huntingtin genes in cells of a non-human mammal which are expressing both said huntingtin genes, and their application for the treatment of Huntington's disease as well as to study Huntington's disease in rodent models