Patent ID: 9441221
Filing Date: 2016-09-13
CPC Classification: A61K,C12N

Claim Text:
1. A compound for inhibiting the expression of a gene of interest comprising: a) at least one nucleic acid molecule comprising an annealing domain operably linked to at least one effector domain, wherein said annealing domain hybridizes to the pre-mRNA of said gene of interest, wherein said effector domain hybridizes to the U1 snRNA of U1 snRNP, wherein said effector domain comprises a sequence having at least 80% identity with the sequence 5′-CAGGUAAGUA-3′ (SEQ ID NO: 1), and wherein said annealing domain is about 10 to about 50 nucleotides in length; and b) at least one targeting moiety, wherein said targeting moiety is operably linked to said nucleic acid molecule.