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. 2022 Oct 9;12(16):7132–7157. doi: 10.7150/thno.77830

Table 3.

Ribozymes and deoxyribozymes used for cancer suppression

Ribozymes
mRNA target Type of cancer Cell culture/animal model/phase of clinical study Nucleic acid modifications Tumor delivery Off-target effects Reference
In vivo (animal models) ft-1 vascular endothelial growth factor (VEGF) numerous human tumor types Cynomolgus monkeys, mice 4 phosphorothioate bonds and an inverted 3'-3' deoxyabasic sugar in one of the recognition arms intravenously,
subcutaneously
None 135
HERZYME
HER-2/neu
breast cancer nude mice 4 phosphorothioate bonds and an inverted 3'-3' deoxyabasic sugar in one of the recognition arms subcutaneously None 136
Clinical studies ft-1 vascular endothelial growth factor (VEGF) breast and colorectal cancer Phase I, multidose phase I/II, phase II 4 phosphorothioate bonds and an inverted 3'-3' deoxyabasic sugar in one of the recognition arms intravenously,
subcutaneously
None 137,138
HERZYME breast cancer Phase I 4 phosphorothioate bonds and an inverted 3'-3' deoxyabasic sugar in one of the recognition arms subcutaneously None 139
Deoxyribozymes
mRNA target Type of cancer Cell culture / animal model / phase of clinical study Nucleic acid modifications Tumor delivery Comments Reference
In vivo
(animal models)
c-jun nodular basal-cell carcinoma primates, mice - intravenously Off-target: activation of inhibitor of caspase-activated deoxyribonuclease and protein kinase C delta 144,145
MMP-9 normal and transgenic mice - None 148
EGR-1 breast carcinoma nude mice - intratumoral None 149
Clinical studies c-jun nodular basal-cell carcinoma phase I - intratumoral None 146
EBV-LMP1 Nasopharyngeal carcinoma phase I phosphorothioate-modified “10-23” DNAzymes intravenous administration None 147