Designer gene therapy may target specific body area

January 20, 2004 | Source: KurzweilAI

Doctors may soon be able to inject genetically engineered “designer” gene therapy intravenously that travels to a specific part of the body, according to Dr. Andrew H. Baker, molecular medicine researcher at the University of Glasgow,

Gene therapy involves inserting the treatment genes into a virus that is either harmless to humans or has had its disease-causing component removed. The virus is then injected or inserted into the body where it “infects” an area with gene therapy.

Baker’s team redesigned a virus called adeno-associated virus (AAV) so that it is not quarantined by the liver, but rather remained in the bloodstream long enough to “infect” specific cells in the body — in this case, the vascular endothelial cells (ECs), which line the inside of blood vessels.

Baker said that AAV is important because it has the potential for long-term gene expression from a single dose and does not cause disease in humans.

Baker said that his results could improve the selectivity, efficiency, and safety of gene delivery to the cardiovascular system.