RNAI INDUCED REDUCTION OF ATAXIN-3 FOR THE TREATMENT OF SPINOCEREBELLAR ATAXIA TYPE 3

摘要

The current invention relates to gene therapy approaches for the treatment of SCA3, in particular RNAi based gene therapy approaches utilizing a total knockdown approach. The inventors provide for selected target regions and/or target sequences for which highly efficient knockdown of the ATXN3 gene expression can be advantegeously obtained in human neuronal cells and in mouse models relevant for SCA3.