Esteban Engel, a viral neuroengineering researcher with Princeton Neuroscience Institute and team have developed a system for switching on the activity of genes that may contribute ultimately to new approaches to improve treatments for a broad range of neurological diseases. At the center of this advancement: the development of new gene promoters—which act like switches to turn on gene expression—that promise to broaden the ability to deliver large genes and keep them active for long periods of time.
Published recently in the journal Molecular Therapy: Methods & Clinical Development, the team developed the “genetic switches” for use in gene therapy—that is the practice of delivering new genes to replace or assist ones that are faulty. Gene therapy is a promising strategy for many diseases, such as disorders that involve the brain, such as Parkinson’s disease and Alzheimer’s disease.
What is the Princeton’s team’s breakthrough?
Engel and the team created new gene promoters that turn on genes after they have been transported into neurons—that is the cells of the brain and nervous system. By adopting attributes of promoters found in another class of viruses, the herpes viruses—which persist for years in the body by establishing a chronic infection in the nervous system—outcomes occupy far less space than existing promoters used in gene therapy. This affords new opportunity to transport larger genes or even multiple genes. Moreover, the new promoters are also longer lasting, and consequently less prone to repression or inactivation than most common promoters—hence the therapeutic genes could be active for long periods of time. These new promoters work with AAV just like other viral and non-viral gene-delivery systems.
Intellectual property & Business Development
Princeton has filed a patent and is seeking outside interests for further development of this asset via the Office of Technology Licensing led by John Ritter.
Esteban Engel, PhD, Principal investigator
Carola J. Maturana, PhD, Senior Author
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