The science of developing nanoparticles as delivery tools for various biologically active agents – genes and drugs – has come a long way. Material science advances have allowed for the construction of nanoparticles with different physicochemical properties, which has, in turn, resulted in remarkable advances, particularly in the area of nucleic acid delivery for the treatment of serious diseases through gene therapy.
A new study by the lab of Dr. Eric Wang from the Massachussetts Institute of Technology and the Department of Molecular Genetics and Microbiology at the University of Florida took the advances in nanoparticles-based delivery one step further.
The authors recognized issues with the way nanoparticles are currently used in delivery – which relies on inefficient in vitro distribution studies which often do not translate well to in vivo settings – and developed a way to screen many PEGylated nanparticles in an in vivo setting, to quickly predict their distribution in vivo.
Titled Barcoded nanoparticles for high throughput in vivo discovery of targeted therapeutics, the study was published last month in the Proceedings of the National Academy of Sciences.
The authors developed chemically-distinct lipid nanoparticles for this study. The nanoparticles carried particular DNA cargo, which the authors termed “barcodes,” which allowed for the rapid screening, via next generation sequencing, after such nanoparticles were administered to mice.
In particular, the authors were interested in observing the exact localization of these nanoparticles – be it lungs, liver or heart.
In this way, they claimed, they could avoid the non-specific delivery of nanoparticles in vitro, which cannot be replicated in many cases, successfully in vivo.
As an example, the authors screened nanoparticles containing, among others, C12-2000, a liver-targeting lipid, and 7C1, a lipid that targets lung.