Gene therapy means putting in a workinggene to a person who has a damaged gene. TheEuropean Commission has approved this method for one particular treatment.[1] The treatment by the productGlybera uses avirus to infect muscle cells with a working copy of the gene. The European Commission has givenGlybera marketing authorisation, which means it can be sold throughout the EU.
One in amillion people have damaged copies of thelipase gene needed to break downfats. Fat builds up in their blood; this leads to pain and inflammation of thepancreas (pancreatitis). It is life-threatening. Up to now, the only way to manage the condition is to have a very low-fat diet.
When used in this way, a virus is avector.[2][3] That means it is a carrier; the gene has been inserted into the viral genome, and the virus sticks it into the human cells. The technique is calledtransfection. This technique is different from thegene knockout technique, which does not use a viral vector.
China was the first country to approve the commercial production of a gene therapy, in 2003. That gene therapy is for head and necksquamous cellcarcinoma (HNSCC)—a cancer that occurs in 10% of new cancers in China. There are 2.5 million new cancer patients every year in China.[4]
Sold under the brand nameGendicine, the world's first commercial gene therapy also uses a virus to insert the gene into the humangenome.
The possibility of gene therapy was talked about as soon as the roles of DNA and RNA were known about. The first specific publication was in 1972.[5]
Many more types of gene therapy are in the trial stage. It is clear that a cycle of events will be part of future clinical medicine: DNAsequence analysis > identify defective genes > fix genes with gene therapy or gene knockout. There are sometimes problems of side-effects; these may be discovered at the trial stage.[6] There is now adatabase which lists all known clinical trials.[7]
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