Patent ID: 8841271
Filing Date: 2014-09-23
Classification: A61K,A61P,C07K,C12N

Abstract:
1. A method for targeted gene repair, comprising contacting the non-repaired target RNA with a phosphorothoiate (PS) containing sequence comprising a deoxynucleotide with RNase H resistant flanking ends; contacting the non-repaired target RNA with an RNA oligonucleotide complex comprising a first oligonucleotide and a second oligonucleotide, said first oligonucleotide comprising a sequence complementary to a repaired target RNA, wherein the RNA sequence of the first oligonucleotide comprises an RNase H-resistant modification, and wherein the RNase H-resistant modification is the addition of a 2-O-methyl moiety, and said second oligonucleotide comprises an RNA sequence complementary to at least 6 nucleotides of the first oligonucleotide at the site in the sequence of the first oligonucleotide which is not complementary to the non-repaired target RNA; and hybridizing said complex to said non-repaired target RNA in the presence of an RNase, wherein a repaired RNA is produced.