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).
Published Version (
Free)
Talk to us
Join us for a 30 min session where you can share your feedback and ask us any queries you have
Schedule a callDisclaimer: All third-party content on this website/platform is and will remain the property of their respective owners and is provided on "as is" basis without any warranties, express or implied. Use of third-party content does not indicate any affiliation, sponsorship with or endorsement by them. Any references to third-party content is to identify the corresponding services and shall be considered fair use under The CopyrightLaw.
