Therapeutic Applications
Modified hammerhead ribozymes are being tested as therapeutic agents. Synthetic RNAs containing sequences complementary to the mutant SOD1 mRNA and sequences necessary to form the hammerhead catalytic structure are being studied as a possible therapy for amyotrophic lateral sclerosis. Work is also underway to find out whether they could be used to engineer HIV-resistant lines of T-cells.Modified hammerhead ribozyme adenoviruses have been shown to be potent in treating cancer both in vitro and in vivo.
The therapeutic use of trans-cleaving hammerhead ribozymes has been severely hampered by its low-level activity in vivo. The true catalytic potential of trans-cleaving hammerhead ribozymes may be recouped in vivo and therapeutic derivatives are likely to complement other nucleic acid hybridizing therapeutic strategies. Already there are hammerhead ribozymes which are close to clinical application.
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“As a science of the unconscious it is a therapeutic method, in the grand style, a method overarching the individual case. Call this, if you choose, a poet’s utopia.”
—Thomas Mann (1875–1955)