RNA interference mediated inhibition of the FCεR1α gene
View Patent ↗The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of FCεR1α gene expression and/or activity, and/or modulate a FCεR1α gene expression pathway. Specifically, the invention relates to doublestranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against FCεR1α gene expression.
1. A double-stranded short interfering nucleic acid (siNA) molecule wherein the siNA is:
wherein:
each B is an inverted abasic cap moiety;
c is 2′-deoxy-2′fluorocytidine;
u is 2′-deoxy-2′fluorouridine;
A is 2′-deoxyadenosine;
G is 2′-deoxyguanosine;
T is thymidine;
U is uridine;
A is 2′-O-methyl-adenosine;
G is 2′-O-methyl-guanosine;
U is 2′-O-methyl-uridine; and
the internucleotide linkages are chemically modified or unmodified.
2. The double-stranded short interfering nucleic acid (siNA) molecule according to claim 1 , wherein the internucleotide linkages are unmodified.
3. A pharmaceutical composition comprising the double-stranded short interfering nucleic acid (siNA) of claim 1 in a pharmaceutically acceptable carrier or diluent.
4. A pharmaceutical composition comprising the double-stranded short interfering nucleic acid (siNA) molecule of claim 1 in an aerosol formulation.