Therapeutic ribozyme compositions

Details Therapeutic ribozyme compositions Therapeutic ribozyme compositions

A - Human Necessities

61

K

A61K 31/70 (2006.01) A61K 47/48 (2006.01) C12N 15/11 (2006.01) C12Q 1/68 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2088917

It is possible to target any RNA molecule for cleavage by RNase P by forming a hybrid region consisting of a short se- quence of base pairs followed ay a terminal 3'-NCCA sequence. In the preferred embodiment, the region is formed by addition of an external guide sequence consisting of a nucleotide sequence complementary to the targeted site which includes a 3'-NNCA, wherein the sequence hybridizes to the targeted RNA to form a short sequence of double-stranded RNA under conditions pro- moting cleavage of the substrate at the nucleotide at the 5' side of the base- paired region by the RNase P. Specificity is deter- mined by the complementary sequence. The sequence is preferably ten to fifteen nucleotides in length and may contain non-com- plementary nucleotides. These embodiments are particularly useful in the treatment of viral diseases and disorders associated with expression of specific proteins from mRNA.

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