Patent ID: 8927519
Filing Date: 2015-01-06
Classification: A61P,C12N,C12Q

Abstract:
1. A method for producing a double stranded small interfering RNA (siRNA) molecule in a mammalian cell, comprising: introducing one or more vectors into a mammalian cell, wherein the one or more vectors comprise (i) a first cassette comprising an RNA pol III promoter operatively linked to a first nucleic acid encoding the sense strand of a double stranded siRNA molecule and (ii) a second cassette comprising an RNA pol III promoter operatively linked to a second nucleic acid encoding the antisense strand of the double stranded siRNA molecule, wherein the first nucleic acid comprises a DNA sequence encoding the sense strand positioned at the first nucleotide of the transcript of the RNA pol III promoter and the second nucleic acid comprises a DNA sequence encoding the antisense strand positioned at the first nucleotide of the transcript of the RNA pol III promoter, wherein the first cassette and the second cassette are in the same vector or are in separate vectors, wherein the double stranded siRNA molecule is specific for a target gene having a target sequence, and allowing transcription of the first and second nucleic acids in the cell, thereby producing the double stranded siRNA molecule, wherein the double stranded siRNA molecule is produced in an amount suitable for inhibiting expression of the target gene.