CPC C12N 15/113 (2013.01) [C12N 15/111 (2013.01); A01K 2207/05 (2013.01); C12N 2310/14 (2013.01); C12N 2310/141 (2013.01); C12N 2310/531 (2013.01); C12N 2320/30 (2013.01)] | 15 Claims |
1. An adeno-associated virus (AAV) vector comprising a sequence encoding a short hairpin RNA (shRNA) operably linked to an RNA polymerase promoter, said shRNA comprising:
a) a first sequence of 21, 22 or 23 nucleotides complementary to a sequence in a target gene, in a sequence other than the mature sequence of miR-451,
b) a second sequence directly following the first sequence, wherein the entire second sequence is fully complementary to the sequence of the first 17 or 18 nucleotides counted from the 5′ end of the first sequence,
wherein the first sequence and the second sequence form the shRNA, and wherein the last 3 or 4 nucleotides of the first sequence form a loop region in the shRNA.
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