RNAi Therapy

A treatment method that destroys "bad" RNA to get rid of "bad" disease-causing protein.

RNA interference (RNAi) therapy involves creating a short piece of gene "sequence" or DNA "letters" that, when injected into the eye, can interact with native RNA and mark them for destruction. This method is useful for destroying "bad" RNA from mutated genes, to prevent production of toxic or harmful proteins (dominant disease). Scientists and clinicians are currently testing combinations of RNAi therapy with gene therapy to also replace the "bad" gene with a "good," or functioning, gene (recessive disease).