Oct. 17, 2006 MIT scientists are reporting synthesis and laboratory tests of a promising new group of degradable polymer delivery vehicles for gene therapy. The polymers are improved versions of materials first described in 2000 that deliver genes efficiently to specific cells.
The lack of a safe, efficient delivery system for DNA has been a major barrier to clinical use of gene therapy. Viruses and various polymer materials have been used in efforts to insert therapeutic genes into diseased cells. However, those systems have disadvantages. Some of the polymer materials, for instance, insert genes into only a small percentage of cells and do not degrade in the body.
Robert Langer and colleagues used laboratory experiments to show that the polymers can deliver DNA to human liver cancer cells. Their report is scheduled for the current (Oct. 15) issue of the weekly Journal of the American Chemical Society.
One polymer has potential for carrying genes to the supply of tiny blood vessels that enables cancer cells to grow and spread. "We are currently testing the targeting effects of this polymer on endothelial cells, and it is envisioned that this delivery system could permit localization and transfection of the tumor vasculature for cancer therapies," the report states.
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