REVERSIBLE SIRNA-BASED SILENCING OF MUTANT AND ENDOGENOUS WILD-TYPE HUNTINGTIN GENE AND ITS APPLICATION FOR THE 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 mutant 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