Scientists seeking a simple and gentle way to provide short-term gene therapy have a new tool: nanoparticles. In a paper published August 30 in Nature Communications, Dr. Matthias Stephan at Fred Hutchinson Cancer Research Center describes nanoparticles he has developed that can streamline the delivery of bundled genetic material to specific cells.
“What we’re doing is ‘hit-and-run’ gene therapy,” a strategy in which a brief change to certain cells can have a permanent therapeutic effect, said Stephan, an immunobioengineer who led the study.
Currently, scientists pursuing gene therapy must choose either targeted approaches that permanently alter cells’ DNA, or short-term approaches that are damaging to cells and can’t be restricted to a particular cell type. Now there is a third option.
The nanoparticles’ ability to gently and temporarily provide gene-editing proteins to specific cells is “really the key” that makes the new approach stand out...
The nanoparticles are formed by bundling synthetic messenger RNA (modified to be safer and more stable than natural RNA) into miniscule packages surrounded by a biodegradable coat. The coat itself is studded with molecules that help the nanoparticles home in on exactly the right cell type.
The researchers showed that hours after the nanoparticles are taken up by target cells, the cells begin churning out proteins based on the new messenger RNA they’ve received. Then, within days, production ceases as the messenger RNA degrades.
Read the full Fred Hutchinson Cancer Research Center article here.