Patent ID: 7737124
Filing Date: 2010-06-15
Classification: A61K,A61P,C12N

Abstract:
1. A method of expressing an siRNA within a cell, the method comprising: transfecting a packaging cell line with a retroviral construct; recovering a recombinant retrovirus from the packaging cell line; and infecting a target cell ex vivo with the recombinant retrovirus; wherein the recombinant retrovirus construct comprises a first RNA polymerase III promoter region, a first RNA coding region, and a first termination sequence; and wherein expression of the RNA coding region results in the down regulation of a target gene or genome transcript; and wherein the first RNA coding region encodes an siRNA comprising SEQ ID NO: 16, wherein the RNA coding region is substantially complementary to a target region that is from 15 to 30 nucleotides in length.