THERAPEUTIC RIBOZYME COMPOSITIONS

摘要

It is possible to target any RNA molecule for cleavage by RNase P by forming a hybrid region consisting of a short sequence of base pairs followed by 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 promoting cleavage of the substrate at the nucleotide at the 5' side of the base-paired region by the RNase P. Specificity is determined by the complementary sequence. The sequence is preferably ten to fifteen nucleotides in length and may contain non-complementary nucleotides. These embodiments are particularly useful in the treatment of viral diseases and disorders associated with expression of specific proteins from mRNA.