Products and compositions
AssigneeSirnaomics, Inc.
InventorDmitry SAMARSKY
Life of this patent
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Abstract
Nucleic acid products and compositions are provided, together with methods for their use, to modulate, in particular, interfere with or inhibit CFB and C5 gene expression.
Claims38
A nucleic acid construct comprising at least:
- (a) a first nucleic acid sequence that is complementary to a first portion of an RNA which is transcribed from a targeted CFB gene;
- (b) a second nucleic acid sequence that is complementary to a second portion of an RNA which is transcribed from a targeted C5 gene;
- (c) a third nucleic acid sequence that is at least partially complementary to said first nucleic acid portion of (a), so as to form a first nucleic acid duplex region therewith;
- (d) a fourth nucleic acid sequence that is at least partially complementary to said second nucleic acid portion of (b), so as to form a second nucleic acid duplex region therewith,
- wherein the first nucleic acid sequence of (a) is directly linked to the fourth nucleic acid sequence of (d) and the second nucleic acid of (b) is directly linked to the third nucleic acid sequence of (c);
- wherein the construct contains labile sites such that subsequent to in vivo administration the construct is cleaved at said labile sites to yield at least first and second discrete nucleic acid targeting molecules that respectively target the RNA portions transcribed from the targeted genes of (a) and (b);
- wherein the
- wherein (i) the first nucleic acid targeting molecule modulates expression of the target gene of (a), and comprises, or is derived from, the first nucleic acid portion of (a), and (ii) the second nucleic acid targeting molecule modulates expression of the targeted gene of (b), and comprises, or is derived from, the second nucleic acid portion of (b).
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The construct according to claim, wherein said first and second nucleic acid sequences each independently have a length of 18 to 21, 18 to 20, or 19 nucleotides.
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Dependencies are inferred from the claim text.