Good news from biomedical research.
The first ever gene therapy was released for the European market by the European Medicines Agency (EMA) and has also been available in Germany since November 1, 2014. This can finally treat the very rare disease lipoprotein lipase (LPL) deficiency (incidence of 1: 1,000,000).
Lipoprotein lipase is a very important enzyme for digesting fatty acids. Patients therefore suffer from “fatty tissue” in the blood (ie high triglyceride serum levels) with very serious complications, such as pancreatitis (inflammation of the Bacuh salivary gland).
Gene therapy works as follows:
an adeno-associated virus of serotype 1 (a normally apthogenic virus) has been genetically modified so that it introduces an intact lipoprotein lipase gene into hepatocytes (liver cells)
and thus leads to the fact that the LPL can be produced again (see Illustration). The triglyceride serum levels of almost all subjects dropped significantly 3 to 12 weeks after the injections.
The only downside: the high price of € 1.1 million for 42 injections that are needed.
For more information you can contact the developer directly: