Esteban Engel: Novel gene-delivery technology for treatment of disease

Posted on January 16, 2020

Esteban Engel and Carola Maturana

A newly developed system for turning on the therapeutic activity of genes could benefit the treatment of a broad range of genetic diseases. Esteban Engel, a researcher in viral neuroengineering in the Princeton Neuroscience Institute, and his team have developed gene promoters, which act like switches to turn on gene expression, to enable the creation of a wide range of gene therapies with long-lasting therapeutic effects.

The team has engineered three new promoters based on promoters that herpesviruses use to establish a chronic infection in the nervous system. They inserted these new promoters into the adeno-associated virus (AAV), which is widely used to deliver therapeutic genes into cells. These promoters occupy far less space than the promoters in use today, allowing the viral vector to carry larger genes. These novel promoters are also less prone to repression or inactivation than most common promoters, so they sustain gene expression for long periods of time. These new promoters work with AAV as well as other viral and non-viral gene-delivery systems. 

Team members: Lynn Enquist, Henry L. Hillman Professor in Molecular Biology; Carola Maturana, postdoctoral research associate; Andrew Esteves, graduate student

Collaborators: Thomas Pisano, Ph.D. 2019; Jessica Verpeut, postdoctoral research associate; Sam Wang, professor neuroscience

Development status: Patent protection is pending. Princeton is seeking outside interest for further development of this technology. Learn more.