Oligonucleotide compounds for targeting huntingtin mRNA
AssigneeUniversity of Massachusetts
InventorsAnastasia Khvorova, Neil Aronin, Julia Alterman
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Abstract
This disclosure relates to novel huntingtin targets. Novel oligonucleotides for the treatment of Huntington's disease are also provided.
Claims41
A vector for inhibiting the expression of HTT gene in a cell, said vector comprising a regulatory sequence operably linked to a nucleotide sequence that encodes a dsRNA molecule substantially complementary to 5′ CAGUAAAGAGAUUAA 3′ (SEQ ID NO:1), 5′ AUAUCAGUAAAGAGA 3′ (SEQ ID NO:2) or 5′ CUCAGGAUUUAAAAU 3′ (SEQ ID NO:3), wherein said dsRNA molecule is between 10 and 35 bases in length.
A dsRNA molecule that is between 15 and 35 base pairs in length, comprising a region of complementarity which is substantially complementary to 5′ CAGUAAAGAGAUUAA 3′ (SEQ ID NO:1), 5′ AUAUCAGUAAAGAGA 3′ (SEQ ID NO:2) or 5′ CUCAGGAUUUAAAAU 3′ (SEQ ID NO:3), wherein the RNA molecule targets an HTT mRNA and comprises at least one modified nucleotide.
Dependencies are inferred from the claim text.