Rnai induced reduction of ataxin-3 for the treatment of spinocerebellar ataxia type 3
OwnerMelvin Maurice EVERS, Pavlina Stefanova KONSTANTINOVA +1inventors
InventorsMelvin Maurice EVERS, Pavlina Stefanova KONSTANTINOVA, Raygene Michaël MARTIER
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Abstract
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.
Claims16
An expression cassette encoding a double stranded RNA comprising a first RNA sequence and a second RNA sequence, wherein the first and second RNA sequence are substantially complementary, wherein the first RNA sequence has a sequence length of at least 19 nucleotides and is substantially complementary to a target RNA sequence comprised in an RNA encoded by a human ATXN3 gene.
Dependencies are inferred from the claim text.