A naked RNA heptamer targeting the human Bcl-2 mRNA induces apoptosis of HL60 leukemia cells

tRNase ZL-utilizing efficacious gene silencing is a gene control technology, which is based on the property that tRNase ZL can cleave any target RNA under the direction of an appropriate small guide RNA (sgRNA). To find therapeutic sgRNAs to cure hematological malignancies, we investigated behavior of heptamer-type sgRNA. We demonstrated that a heptamer, mh1(Bcl-2), which targets the human Bcl-2 mRNA, can be taken up by cells without any transfection reagents and that it can induce apoptosis of the leukemia cells. Mouse xenograft experiments showed that a median survival of the mh1(Bcl-2)-treated mice was longer than that of the control mice.

► TRUE gene silencing is a gene control technology based on sgRNA-directed specific mRNA cleavage by tRNase ZL.
► Behavior of heptamer-type sgRNA was investigated to find therapeutic sgRNAs to cure hematological malignancies.
► A heptamer, mh1(Bcl-2), which targets the human Bcl-2 mRNA, can be taken up by cells without any transfection reagents.
► mh1(Bcl-2) can induce apoptosis of HL60 leukemia cells.
► In mouse xenograft experiments, a median survival of the mh1(Bcl-2)-treated mice is longer than that of the control mice.