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US 2021/0330813 A1Published application
Rnai induced c9orf72 suppression for the treatment of als/ftd
OwnerRaygene Michaël MARTIER, Pavlina Stefanova KONSTANTINOVAinventors
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Abstract
The present invention provides for specific target RNA sequences that can in particular be applied in RNAi based gene therapy approaches for the treatment of ALS and/or FTD. These specific target RNA sequences were found by selecting target RNA sequences that were conserved in the C9ORF72 target RNA sequence and were shown to provide for efficient silencing. Also are provided combinations of target RNA sequences that are useful in the treatment of ALS and/or FTD.
Claims15
An expression cassette encoding a double stranded RNA comprising:
- a first RNA sequence, wherein the first RNA sequence has a sequence length of at least 19 nucleotides and is substantially complementarity to SEQ ID NO. 32 and to a target RNA sequence comprised in an RNA encoded by a human C9orf72 gene, and
- a second RNA sequence,
- wherein the first and second RNA sequence are substantially complementary.
Dependencies are inferred from the claim text.