Gene therapy works by inserting modified genes to treat an illness or condition into a virus that has been made harmless to humans.
But injecting these gene-carrying viruses into a person's bloodstream has been a problem, because most of the virus ends up in the liver, which naturally isolates foreign bodies and washes them out of the blood. That makes it difficult to deliver enough of the new genes to the part of the body that needs assistance.
New research shows a form of gene therapy capable of entering the bloodstream and traveling directly to the part of the body that needs treatment may be on the horizon.
Scientists from the University of Glasgow in Scotland have now developed a "designer" gene therapy that bypasses the liver. The new therapy uses an adeno-associated virus, or AAV, which has been redesigned to avoid capture by the liver long enough to get to the part of the body needing the new genes.They've tested two forms of the virus in the laboratory and in mice, using it to deliver new genes to vascular endothelial cells, which are the cells that line the inside of blood vessels. These cells play a major role in many cardiovascular abnormalities. Researchers redesigned the virus by modifying two small proteins called peptides.
More work will be needed before the therapy can be tried in humans, but researchers believe these findings hold promise for the future.