DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION

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United States of America Patent

APP PUB NO 20150315579A1
SERIAL NO

14616595

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Abstract

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The present invention provides double stranded compositions wherein the first strand is modified to have a particular motif and the second strand is modified a selected motif. The motifs are defined by positioning of differentially modified nucleosides wherein at least the sugar moieties are different. More particularly, the present compositions comprise an antisense strand that is modified to have a positional/full motif and the sense strand is modified to have an alternating motif, a hemimer motif, a blockmer motif, a gapped motif, a positional motif, a positional/full motif or a fully modified motif. Each strand further comprises one or more phosphorothioate internucleoside linkage. The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In preferred embodiments the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.

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Patent Owner(s)

Patent OwnerAddress
IONIS PHARMACEUTICALS INC2855 GAZELLE COURT CARLSBAD CA 92010

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Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Allerson, Charles San Diego, US 51 2740
Bhat, Balkrishen Carlsbad, US 173 7460
Kinberger, Garth A San Diego, US 24 357
Marcusson, Eric G San Francisco, US 63 1332
Prakash, Thazha P Carlsbad, US 184 5389
Swayze, Eric E Encinitas, US 355 10308

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