Showing posts with label Adeno. Show all posts
Showing posts with label Adeno. Show all posts

May 7, 2013

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.