Hypercholesterolemia

Gene therapy breakthrough offers hope to patients with inherited disease

Published in Gene Therapy Weekly, June 13th, 2002

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 cholesterol 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...

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Source: Gene Therapy Weekly (2002-06-13)

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