Gene Therapy Could Soon Be Approved In Europe 44
another random user writes
"According to the BBC, 'Europe is on the cusp of approving a gene therapy for the first time, in what would be a landmark moment for the field. ... The European Medicines Agency has recommended a therapy for a rare genetic disease which leaves people unable to properly digest fats. The European Commission will now make the final decision. The idea of gene therapy is simple: if there is a problem with part of a patient's genetic code then replace that part of the code. The reality has not been so easy. In one gene therapy trial a U.S. teenager, Jesse Gelsinger, died, and other patients have developed leukaemia. There no gene therapies available outside of a research lab in Europe or the U.S.' They have considered the use of Glybera to treat lipoprotein lipase deficiency, which leads to fat building up in the blood, abdominal pain and life-threatening pancreatitis (inflammation of the pancreas). 'The therapy uses a virus to infect muscle cells with a working copy of the gene.'"
Re:reaction (Score:5, Interesting)
A few years ago, Gattaca was rated the most realistic sci-fi movie by NASA. Keep that in mind everyone.
Re:Rare? (Score:2, Interesting)
You would be insane to use a fully working virus - Instead you package your pay-lode in viral proteins either within a partly functional virus, or as a replacement for a non functional one. For safety reasons the second is always preferable. The first is more risky but you would usually use a virus with no transmission ability outside of the cell line you bread it in, or in the worst case at least no ability to spread between humans. Note that neither of the TFAs differentiate at least as far as I can say so I had to look it up, and normally this info is skipped or even unintentionally lied about in the media due to ignorant journalists.
In this case "most therapeutic genes require the complete replacement of the virus's 4.8 kilobase genome"[1] as it uses a Adeno-associated virus vector[2], so likely it can not spread, this should also be the case due to the normal modifications made before use as a vector. This mean that it will act closer to a genetic drug than a actual vius, affecting the cells it enters permanently but only ever half of the offspring (does not integrate can not divide or piggyback on genomic division).
[1]http://en.wikipedia.org/wiki/Adeno-associated_virus#Advantages_and_drawbacks
[2]http://www.ema.europa.eu/ema/index.jsp?curl=pages/news_and_events/news/2012/07/news_detail_001574.jsp - "Glybera uses an adeno-associated virus vector as the delivery vehicle to add working copies of the LPL gene into muscle cells to enable production of the enzyme in the cells."