Scientists Develop Gene Silencing DNA Enzyme that can Target a Single Molecule
Published:26 May2023    Source:University of California - Irvine

Researchers from the University of California, Irvine have developed a DNA enzyme -- or DNAzyme -- that can distinguish between two RNA strands inside a cell and cut the disease-associated strand while leaving the healthy strand intact. This breakthrough "gene silencing" technology could revolutionize the development of DNAzymes for treating cancer, infectious diseases and neurological disorders.


DNAzymes are nucleic acid enzymes that cut other molecules. Through chemistry, UCI's team developed the Dz 46 enzyme. The benefit of the Dz 46 enzyme is that it can identify and cut a specific gene mutation, offering patients an innovative, precision medicine treatment. The DNAzyme resembles the Greek letter omega and acts as a catalyst by accelerating chemical reactions. The "arms" on the left and right bind to the target region of the RNA. The loop binds to magnesium, and folds and cuts the RNA at a very specific site.


“We solved that problem by re-engineering the DNAzyme using chemistry to reduce its dependency on magnesium and did so in such a way that we could maintain high catalytic turnover activity,” Chaput said, UCI professor of pharmaceutical sciences. The researchers and UCI have filed provisional patent applications on the chemical composition and cleavage preference of Dz 46.