1025. Therapeutic Effect of a Self-Cleaving Hammerhead Ribozyme in a Mouse Model of Osteogenesis Imperfecta

Abstract

Hammerhead ribozymes are small catalytic RNA molecules that can be targeted to any RNA molecule containing a putative cleavage site, and can selectively eliminate mutant gene products producing dominant negative effects, such as those found in severe variants of osteogenesis imperfecta (OI). We recently reported that self-cleaving multimeric hammerhead ribozymes targeted to a truncated COL1A1 transcript are extremely effective in cellulo (Peace, B. E., et al.; “Endogenously expressed multimeric self-cleaving hammerhead ribozymes ablate mutant collagen in cellulo”, Molecular Therapy, 2005).