Double-stranded RNA (dsRNA) and method of use for inhibiting expression of a fusion gene

A ribonucleic acid (RNA) having a double stranded structure comprising a single-self complementary RNA strand, wherein the RNA comprises a nucleotide sequence of between about 19 and about 24 nucleotides in length which is substantially identical to at least a part of a target gene in a mammalian cell, and wherein the target gene comprises a fusion site.

A method for inhibiting the expression of a target gene in a mammalian cell, the method comprising:

• (a) introducing into the cell an RNA comprising a double stranded structure having a nucleotide sequence which is substantially identical to at least a part of a target gene in the mammalian cell, wherein the nucleotide sequence is between about 19 and about 24 nucleotides in length, and wherein the target gene comprises a fusion site;
• and
• (b) maintaining the cell produced in step (a) under conditions and for a time sufficient to obtain degradation of mRNA of the target gene, thereby inhibiting expression of the target gene in the cell.

A method for treating a mammal having a disease caused by the expression of a fusion gene which results from a chromosomal aberration, comprising administering to the mammal an RNA comprising a double stranded structure having a nucleotide sequence which is substantially identical to at least a part of a target gene, wherein the nucleotide sequence is between about 19 and about 24 nucleotides in length, and wherein the RNA inhibits the expression of the target gene.

A method of using an RNA to inhibit the expression of a target gene in a mammalian cell, the RNA comprising a double stranded structure having a nucleotide sequence which is substantially identical to at least a part of a target gene in the mammalian cell, wherein the nucleotide sequence is between about 19 and about 24 nucleotides in length, and wherein the target gene comprises a fusion site.

A pharmaceutical composition comprising RNA comprising a double stranded structure having a nucleotide sequence which is substantially identical to at least a part of a target gene in the mammalian cell, wherein the nucleotide sequence is between about 19 and about 24 nucleotides in length, and wherein the target gene comprises a fusion site, together with a pharmaceutically acceptable carrier.