In a demonstration that could help pave the way for gene therapies with fewer side effects, several human cell types have been genetically modified with protein nanoparticles designed at University of Michigan Engineering and Michigan Medicine. Gene therapy has been enormously successful for treating disorders of the blood, including sickle cell disease and leukemia. However, using a virus as a vector for treatment can create unwanted side effects, such as secondary cancers and immune system overreactions. With the nanoparticles, the research team aims to develop a safer method for delivering gene therapies.

In a proof-of-concept experiment, the researchers used nanoparticles to modify several types of human cells. They made human liver cancer cells, kidney cells and immune cells glow green by giving them genes for green fluorescent protein. The cells activated the new genes after they engulfed and digested the nanoparticles, releasing the DNA or messenger RNA packed inside. The work is published in the journal Advanced Materials.

"There are a lot of diseases where a protein is missing or dysfunctional due to a single mutation, and we can definitely correct for that by introducing a new gene," said Joerg Lahann, the Wolfgang Pauli Collegiate Professor of Chemical Engineering, director of the U-M Biointerfaces Institute and the corresponding author of the study.

"Typically, this is done with viruses, but the viruses can be toxic and activate the immune cells. So there has been a push in the field to replace virus-based gene editing strategies."