Compositions and methods for inhibiting expression of mutant EGFR gene

摘要

The invention relates to a double-stranded ribonucleic acid (dsRNA) targeting a mutant Epidermal Growth Factor Receptor (EGFR), and methods of using the dsRNA to inhibit expression of mutant EGFR.

主权项

1. A method of treating glioblastoma multiforme (GBM), the method comprising administering to a human in need of such treatment a therapeutically effective amount of a first double-stranded ribonucleic acid (dsRNA) and a second dsRNA,
wherein the first dsRNA comprises a sense strand and an antisense strand, wherein the antisense strand comprises a region of complementarity which is 19-24 nucleotides in length and which is fully complementary to at least a part of an mRNA encoding a deltaEGFR comprising nucleotides 1-334 and 1136-5616 of the nucleotide sequence of SEQ ID NO: 1, wherein the sense strand and the antisense strand form a duplex structure consisting of 15-30 base pairs; and wherein the second dsRNA comprises a sense strand and an antisense strand, wherein the antisense strand comprises a region of complementarity which is 19-24 nucleotides in length and which is fully complementary to at least a part of an mRNA encoding an IL-6 comprising the nucleotide sequence of SEQ ID NO: 274, wherein the sense strand and the antisense strand form a duplex structure consisting of 15-30 base pairs.