Engineered Virus With Dual Protease Key System Opens to Release Gene Therapy

Viruses cause many diseases but can also serve as vectors for delivery of genetic cargo for therapeutic purposes. Rice University researchers have now modified the adeno-associated virus, commonly used to deliver gene therapy, to work like a lock box that opens itself up only in the presence of two different chemical “keys”.

The virus responds to proteases, enzymes that break down other proteins, opening up and releasing the cargo only when both of the markers are present. By selecting which proteases unlock the virus, a new form of therapy may develop that allows doctors to precisely tune where gene delivery happens.

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“We were looking for other types of biomarkers beyond cellular receptors present at disease sites,” [Junghae Suh, bioengineer at Rice] said. “In breast cancer, for example, it’s known the tumor cells oversecrete extracellular proteases, but perhaps more important are the infiltrating immune cells that migrate into the tumor…