Erythrocyte Ghosts for Gene Therapy

One of the main challenges faced by researchers working on drug delivery and gene therapy is how to effectively target the therapy to the area that needs it and to ensure that the drug stays in the body for an extended period of time. Various methods have been tried, from straight injection of DNA to cationic vectors to improve the survival time of the DNA in the blood. However, these methods are less than ideal when it comes to gene therapy. For example, the vector approach uses nonviral vectors, which are actually toxic to the body. To various teams of researchers, it seems that a simple answer to this problem has presented itself in the form of erythrocytes, or red blood cells.

Erythrocytes are biocompatible, relatively long lived, are found in abundance in the body, and travel all over the body. In this article, a team of researchers was able to manipulate the erythrocyte membrane in order to encapsulate plasmid DNA within the cells. These loaded cells, or “erythrocyte ghosts”, were then tested in circulation in mice and the life of the DNA was compared to the life of DNA in other methods of delivery. Researchers observed that the plasmid DNA-loaded erythrocyte ghosts survived and remained active in vivo much longer than that of plain injected plasmid DNA. They also reinforced the idea that targeting can be achieved through the blood stream and proved the viability of erythrocyte ghosts as carrier agents for therapeutic drugs in the body.

This article intrigued me because of its minimally invasive approach to solving a problem. Using erythrocyte ghosts works in sync with the body’s natural systems to fix the problem in lieu of harsher methods that could cause more damage to the body. It seems to me that a smart move in medicine is to make use of the body’s own capabilities in medical treatments, which is precisely what the erythrocyte ghost method does. The use of erythrocyte ghosts could have a far reaching impact past just gene therapy. In fact, there is research being conducted in our own biomedical engineering department for other uses of erythrocyte ghosts (see link at bottom). The potential for erythrocyte ghosts is immense and is definitely a unique approach to solving problems in the body.

http://www.nature.com/gt/journal/v11/n5/full/3302180a.html

http://www.nature.com/nrd/journal/v2/n9/full/nrd1189.html

http://biomed.tamu.edu/meissnerlab/Research.html