Gene Therapy Virus

In 2012 the European union authorized UniQure to use Glybera gene therapy for commercial use. The medicine sends a virus into your body, containing the correct genetic code. The therapy, developed by UniQure uses a virus to infect muscle cells with a working copy of the gene. Once the virus infects muscle cells, the correct code overwrites the bad DNA.

Glybera is used to treat lipoprotein lipase deficiency. One in a million people have damaged copies of a gene which is essential for breaking down fats. It means fat builds up in the blood leading to abdominal pain and life-threatening inflammation of the pancreas, pancreatitis.

A few years ago, three academic groups showed that AAV2, another adeno-associated virus, can correct a rare form of inherited blindness, by targeting a certain cell type within the retina.