Glybera - Gene Therapy

[bigstar]

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]

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 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, which (it is hoped) will fly under the radar of the body's immune system.