Chances are you haven’t heard much about lipoprotein lipase deficiency (LPLD), a disease that leads to pancreatitis. This rare disease, however, is at the center of world-changing medical advances.
Last week the European Union approved a gene therapy treatment for LPLD–and this marks the first time any medical treatment that rewrites a patients’ DNA has been approved for commercial use.
The treatment, called Glybera, will be released by Dutch firm uniQure in the second half of 2013. Glybera will be administered to patients by specially trained doctors at a limited number of European hospitals. Patients receiving treatment have their DNA altered by a series of injections into their leg muscles, which helps normalize the metabolism of fat particles carried in the blood. LPLD prevents sufferers from properly metabolizing these particles, leading to a host of side effects including pancreatitis.
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They have to extremely careful with these treatments. Past attempts to treat other diseases produced cancers in most patients when the introduced sequences landed in oncogenic-inducing regions of their genomes.