The new delivery platform works efficiently in cell models, and, with further development, could open up a new class of delivery methods for a wide range of molecular medicinesincluding those for gene editing and gene replacement. Existing delivery vehicles for this therapeutics can be inefficient and randomly integrate into the genome of cells, and some can stimulate unwanted immune reactions. SEND has the promise to overcome these limitations, which could open up new opportunities to deploy molecular medicine.

"The biomedical community has been developing powerful molecular therapeutics, but delivering them to cells in a precise and efficient way is challenging," said CRISPR pioneer Feng Zhang, senior author on the study, core institute member at the Broad Institute, investigator at the McGovern Institute, and the James and Patricia Poitras Professor of Neuroscience at MIT. "SEND has the potential to overcome these challenges." Zhang is also an investigator at the Howard Hughes Medical Institute and a professor in MIT's Departments of Brain and Cognitive Sciences and Biological Engineering.

At the center of SEND is a protein called PEG10, which normally binds to its own mRNA and forms a spherical protective capsule around it. In their study, the team engineered PEG10 to selectively package and deliver other RNA. The scientists used SEND to deliver the CRISPR-Cas9 gene editing system to mouse and human cells to edit targeted genes. This study shows that there are probably other RNA transfer systems in the human body that can also be harnessed for therapeutic purposes. It also raises some really fascinating questions about what the natural roles of these proteins might be.

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John
EditorialAssistant
Immunogenetics Open Access