Researchers at Penn Engineering have developed mvGPT, an advanced genetic editing tool that combines gene editing and regulation in one platform. This breakthrough could revolutionize the treatment of genetic diseases.
Researchers at the University of Pennsylvania’s School of Engineering and Applied Science (Penn Engineering) have unveiled a revolutionary genetic editing tool known as minimal versatile genetic perturbation technology (mvGPT). This technology, introduced in a paper published in Nature Communications, has the transformative potential to simultaneously edit multiple genes and regulate their expression, offering new hope for the treatment of complex genetic diseases.
“Not all genetic diseases are solely caused by errors in the genetic code itself,” senior author Sherry Gao, Presidential Penn Compact Associate Professor in Chemical and Biomolecular Engineering and Bioengineering, said in a news release. “In some cases, diseases with genetic components — like type I diabetes — are due to how much or little certain genes are expressed.”
Traditionally, separate tools were needed for gene editing and gene expression regulation, presenting logistical and technical challenges in treating diseases with multiple genetic components.
The innovative mvGPT technology merges these functions into a single platform.
“We wanted to build a single platform that could precisely and efficiently edit DNA as well as upregulate and downregulate gene expression,” co-first author Tyler Daniel, a doctoral student in the Gao Lab, said in the news release.
mvGPT has shown unprecedented precision in independent multi-gene editing and regulation, which was previously unattainable.
“Each task functions independently. It’s as if we took a car with a faulty navigation system and fixed the bug in that system while simultaneously turning up the volume on the stereo and turning down the air conditioning,” Daniel added.
In tests on human liver cells, mvGPT successfully edited mutations causing Wilson’s disease while simultaneously upregulating genes linked to type 1 diabetes treatment and repressing those associated with transthyretin amyloidosis. Remarkably, the platform achieved these tasks with high precision and efficiency.
One of the most compelling aspects of mvGPT is its potential to treat a wide range of genetic conditions.
The team’s success in human cells is just the beginning. The researchers plan to extend their testing to animal models and explore applications for diseases with genetic components, including cardiovascular diseases.
“The more advanced our tools become, the more we can do to treat genetic diseases,” Gao added.
The genetic perturbation technologies described in the study are also pending patent approval (application number 63/712,648). This research involved contributions from Rice University and Baylor College of Medicine, reflecting a strong collaborative effort.
By simplifying the process of delivering genetic tools into cells and enabling multifaceted genetic modifications, mvGPT stands to radically change how scientists and doctors approach genetic diseases. The potential for simpler, more effective treatments marks a significant milestone in genetic engineering.
As the technology advances and is tested in more complex systems, mvGPT could become a cornerstone in the treatment and understanding of numerous genetic conditions, ushering in a new era of precision medicine.