RNAi induced huntingtin gene suppression
AssigneeuniQure IP B.V.
InventorsPavlina Stefanova Konstantinova, Jana Miniariková
Life of this patent
Estimating the term…
Abstract
The present invention provides for 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 SEQ ID NO. 1. Said double stranded RNA is for use in inducing RNAi against Huntingtin exon 1 sequences. The double stranded RNA of to the invention was capable of reducing neuronal cell death and huntingtin aggregates in an animal model.
Claims18
A method of reducing expression of an RNA transcript comprising SEQ ID NO:1 in a neuron, method comprising introducing into the neuron 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 comprises SEQ ID NO:3, SEQ ID NO:4, SEQ ID NO:5, SEQ ID NO:6, or SEQ ID NO:7.
A method of treating a subject with Huntington's disease or a subject that is genetically predisposed for Huntington's disease who does not yet show symptoms of Huntington's disease, comprising administering to a subject with Huntington's disease an effective amount of a viral vector 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 comprises SEQ ID NO:3, SEQ ID NO:4, SEQ ID NO:5, SEQ ID NO:6, or SEQ ID NO:7.
Dependencies are inferred from the claim text.