by Dan Roberts
Researchers have developed a new tool for gene therapy that significantly increases gene delivery to cells in the retina compared to other carriers and DNA alone. The study was published in the January 2010 issue of The Journal of Gene Medicine by Tufts University School of Medicine and the Sackler School of Graduate Biomedical Sciences at Tufts (Rajendra Kumar-Singh, PhD, senior author).
A peptide called PEG-POD provides a safe and effective vehicle for transferring DNA into cells without using a virus, currently the most common means of DNA delivery. According to Dr. Kumar-Singh, PEG-POD protects DNA from damage in the bloodstream, allowing for gene therapy treatments that can be administered through an IV and directed to many other parts of the body.
The researchers used an in vivo model to compare the effectiveness of PEG-POD with two other carriers (PEG-TAT and PEG-CK30) and a control (injections of DNA alone). They found that gene expression in specimens injected with PEG-POD was far more effective than the other two carriers at 215 times greater than the control.
Read SP, Cashman SM, Kumar-Singh R. A poly(ethylene) glycolylated peptide for ocular delivery compacts DNA into nanoparticles for gene delivery to post-mitotic tissues in vivo. The Journal of Gene Medicine, 2010; 12 (1): 86 DOI: 10.1002/jgm.1415