Human Proteins Enable Researchers To Deliver Medication Directly To The Cells

Researchers at MIT and other institutions have created a programmable system based on human proteins to deliver gene editing and molecular therapeutics directly into cells. The system developed by the researchers is called SEND and is programmable to encapsulate and deliver various RNA cargoes directly to the cell. SEND leverages natural proteins found in the human body to form particles similar to a virus that bind RNA.The method generates less of an immune response than similar delivery systems by harnessing proteins occurring naturally in the human body. Researchers found the delivery system works efficiently in cell models, and they believe additional development could result in a new class of delivery methods for a range of molecular medications. For example, the system could be used for gene editing or replacement.

While there are existing delivery platforms for gene editing and replacement, they can be inefficient. One big downside to existing systems is they randomly integrate into the cell's genome, stimulating immune reactions. SEND could overcome the issues surrounding current delivery systems. SEND stands for Selective Endogenous eNcapsidation for cellular Delivery.

One of the core components for SEND is the PEG10 protein that in the human body binds to its own mRNA to form a protective capsule around it. For the study, researchers engineered the PEG10 package to deliver other RNA. They were able to deliver the CRISPR-Cas9 gene editing system into mouse and human cells. Once in the cells, that editing system was able to edit targeted genes.

Researchers in the study note there are likely other RNA transfer systems within the human body that could also be used for therapeutic purposes. The protein harnessed in this study, PEG10, exists naturally in the human body and comes from a virus-like genetic element that integrated itself into our ancestors millions of years ago. The body transformed protein into one of many important for life as we know it.