Article
Endogenously expressed multimeric self-cleaving hammerhead ribozymes ablate mutant collagen in cellulo.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Jul 2005
Peace Belinda E, Florer Jane B, Witte David, Smicun Yoel, Toudjarska Iva, Wu George, Kilpatrick Michael W, Tsipouras Petros, Wenstrup Richard J
Abstract excerpt
Hammerhead ribozymes are small catalytic RNA molecules that can be targeted to any RNA molecule containing a putative cleavage site. We developed a vector (pCOLZ) that uses the COL1A1 promoter to drive expression of a self-cleaving multimeric ribozyme (M8Rz547) and its monomeric counterpart (Rz547). The ribozymes were stably coexpressed in MC3T3-E1 osteoblasts expressing a truncated COL1A1 target transcript. The...
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