Birmingham researchers have developed a gene therapy system that could lead to a cure for people with high blood cholesterol - and may have broader applications in the development of gene therapy for other diseases.
One of the biggest problems facing the development of gene therapy, where doctors aim to replace faulty genes leading to disease expression with new copies, is getting the replacement genes into the right cells in the body. So far, systems using viruses to deliver genes to cells have proved inefficient.
But a team at Birmingham University, led by senior lecturer Leonard Seymour, is using polymers - substances formed by the linkage of a large number of smaller molecules called monomers - to deliver genes into liver cells. Early results show that polymers are non-toxic, unlike viral systems, and are more effective in carrying replacement genes to the target site.
They hope this could be used to help patients with high cholesterol caused by a faulty gene in their liver cells. The cells need the gene to make a protein which controls cholesterol levels in the blood.
Dr Seymour said: 'If the research continues to be successful we should see major advances within a few years.'
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