Patent ID: 9175287
Filing Date: 2015-11-03
Classification: A01K,A61P,C12N

Abstract:
1. An adeno-associated viral (AAV) vector comprising an isolated nucleic acid molecule comprising a regulatory sequence operably linked to a nucleic acid sequence that encodes an engineered ribonucleic acid (RNA) precursor which is processed to form a small interfering ribonucleic acid (siRNA) comprising an antisense strand complementary to a sequence of a messenger RNA (mRNA) of a target gene and a sense strand having a sequence complementary to the antisense strand, wherein the precursor comprises (i) a first stem portion consisting of a sequence of about 18 to about 40 nucleotides that is complementary to a sequence of the of the mRNA of the target gene; (ii) a second stem portion consisting of a sequence of about 18 to about 40 nucleotides that is sufficiently complementary to the about 18 to about 40 nucleotide sequence of the first stem portion to hybridize with the first stem portion to form a duplex stem; and (iii) a loop portion consisting of 2 to 9 nucleotides that connects the two stem portions.