Deoxyribozymes: cleaving a path to clinical trials

Deoxyribozymes (DNAzymes) comprise an exciting class of nucleic acid molecules that are capable of specific cleavage of target mRNA. Recent reports attest to the potential of this class of molecules in cell culture and preclinical studies, where DNAzymes exhibit the ability to silence disease-associated genes (mainly those associated with cardiovascular disease and cancer). Rigorous testing in preclinical studies is now required before this relatively new entity enters Phase I clinical trials.