Double-stranded RNA (dsRNA) and method of use for inhibiting expression of a fusion gene
AssigneeAlnylam Europe AG, a German corporation
InventorsOlaf Heidenreich, Hans-Peter Vornlocher, Roland Kreutzer, Stefan Limmer
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Abstract
The present invention relates to the specific inhibition of expression of a fusion gene in mammals using a short double stranded RNA. The dsRNA is approximately 19-24 nucleotides in length, and has a nucleotide sequence which is complementary to at least a part of the target gene. The dsRNAs of the present invention are useful for treating diseases caused by chromosomal aberrations, particularly malignant diseases such as lymphoma and leukemia.
Claims17
1-67. (canceled)
A method for inhibiting the expression of a target fusion gene in a mammalian cell, comprising the steps of:
- a) introducing into a mammalian cell a dsRNA, said dsRNA comprising two separate non-linked RNA strands, a S1 strand and a complementary RNA strand, wherein each RNA strand consists of from 20 to less than 30 nucleotides in length and said S1 strand is complementary to the fusion junction of said target fusion gene and comprises at least 7 nucleotides an each side of said fusion junction, and
- b) maintaining the cell produced in step (a) under conditions and for a time sufficient to obtain degradation of mRNA of said target fusion gene, thereby inhibiting expression of said target fusion gene in the cell.
A method for inhibiting the expression of an AML-1/MTG-8 fusion gene in a mammalian cell, comprising the steps of:
- a) introducing into a mammalian cell a dsRNA, said dsRNA comprising two separate non-linked RNA strands, a S1 strand and a complementary RNA strand, wherein each RNA strand consists of from 20 to less than 30 nucleotides in length and said S1 strand is complementary to SEQ ID NO:
- 2, and
- b) maintaining the cell produced in step (a) under conditions and for a time sufficient to obtain degradation of mRNA of said target fusion gene, thereby inhibiting expression of said target fusion gene in the cell.
Dependencies are inferred from the claim text.