Some people’s cells have genes that are malfunctioning, or missing altogether, leading to a variety of serious diseases. Patients with these problems can be treated by delivering new, functional genes into their cells using carefully engineered viruses, which can be built to carry the desired genetic material into the body and incorporate it into the patient’s cells.
Unfortunately, the human body’s defenses respond to the engineered viruses just as they would to natural ones: as if they were a potentially deadly threat. These defenses have presented a substantial challenge to effective gene therapy: how to deliver the precious genetic cargo to cells without activating the body’s natural defenses.
Our transformative technology overcomes this most significant challenge. The breakthrough came by identifying the precise reason that viruses were setting off the body’s alarm bells. By removing the chemical signatures that were discovered to be the culprits, viruses could be designed to deliver the curative genes without activating the body’s immune system.