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View Full Version : Glybera - Gene Therapy



bigstar
11-05-2012, 07:15 AM
http://www.uniqure.com/products/glybera/

Well i came across this article on a Greek Forum about Cf, but im not sure what does this means for us!

MaddiesFamily
11-05-2012, 03:40 PM
im not sure what does this means for us!
Possibly not too much. One of the principal challenges for gene therapy is finding a vector which can be used to administer repeated doses of the corrected gene. There is some uncertainty as to whether adeno-associated virus vectors will trigger an immune response when repeated. Looking at the information relation to Glybera, it seems that it has long-lasting effects from a single-dose (so that any problems with repeated dosing would not arise).
The Phase 2 gene therapy trial (http://www.cfgenetherapy.org.uk/clinical/product.html)now under way in the UK uses a liposome vector (effectively a fat globule) to get around the problems experienced with virus vectors. Work is also underway on a Wave 2 product using a lentiviral vector (http://www.cfgenetherapy.org.uk/mfia2011/lentiviral.html), which (it is hoped) will fly under the radar of the body's immune system.