New research published in BMC Molecular Biology explains how a new technique for introducing genes into mammalian cells using the virus responsible for warts could be a major step forward in developing gene therapy treatments for people with familial hypercholesterolemia (FH), a genetic disease that affects around 12 million people worldwide.
People with FH have a genetic defect that prevents their liver cells from absorbing chlolesterol in the form of low density lipoprotein (LDL). This leads to high levels of cholesterol in the bloodstream increasing the risk of heart disease. It is estimated that about half of men and a third of women with FH suffer a heart attack by the age of 60.
FH occurs as a result of a single defective gene that codes for a LDL receptor in the liver. Many single gene defects like the one that causes FH are candidates for gene therapy, a medical treatment used to repair or replace faulty genes (this is because it is much easier to repair one gene that causes a disease than it is to tackle other genetic diseases that involves several faulty genes).
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