New gene therapy delivery system

Dr.David Liu with his colleagues at Harvard Medical School have developed a technique that allows administration of specific proteins which can make changes to the genome into cells. Positively charged cationic lipids are used for delivery of proteins.

The authors reported that their proposed technology has been successfully tested not only in cell cultures but also in animals. The scientists were able to modify genes in hairlike cells located in the inner ear of mice. Damage to these cells associated with both external and genetic factors, leads to hearing loss.

Delivery technique, developed by researchers was much more effective than other delivery mechanisms. They used cationic lipids wich were positively charged and were able to bind to negatively charged nucleic acids. This leads in the formation of liposomes, by which the contents are delivered into the cell.

Using the same cationic lipids authors were also able to administrate proteins into cells. The researchers note that the effectiveness of this delivery system was about 1000 times higher than the more common approach of using positively charged peptides or proteins.

Scientists note that the use of protein for changes in the genome, more preferable than the use of DNA for the same purpose. They believe that a new delivery system will be widely used in clinical practice and help in the fight against diseases difficult to treat in future.

Date: 11\11\2014