Monthly Research Publication Highlight details a novel method to quantify the intracellular uptake and trafficking of gene therapy nanoparticles

The UK College of Pharmacy Research Publication Highlight for February, 2017 is titled “Rapid and facile quantitation of polyplex endocytic trafficking” and was published in the The Journal of Controlled Release.

The project was completed by investigators from the University of Illinois and the University of Kentucky Colleges of Pharmacy and Engineering.  The lead author is Dr. Mihael Lazebnik, a former student currently working in the Early Stage Cell Culture unit at Genentech, in the laboratory of Dr. Dan Pack, the Ashland Inc. Chair in Chemical Engineering who holds joint appointments in Pharmaceutical Sciences and Chemical and Materials Engineering.

A number of technologies to express modified genes or alter the expression of endogenous genes have been shown to be effective in the treatment of disease in preclinical models.  However, a major impediment to the translation of these technologies to patients is the delivery of these gene-modifying approaches to the appropriate tissues and cells.  Further, how these delivery vehicles are taken-up and transported within the cell to access the nucleus is unclear.  The authors of the present study developed a fast and quantitative method to track uptake, accumulation and intracellular transport of nanoparticle deliver systems for gene therapy that will allow investigators to optimize various nucleic acid delivery vehicles for nuclear targeting and efficacy.

“The development of this analytical tool is an important step forward in the translation of gene therapies shown to be effective in the lab to the clinical setting where they can be used in the treatment of a variety of diseases” said Greg Graf, Assistant Dean for Translational Research.