With the recent gene therapy successes with Hemophilia B, how long before we get attempts at Hemophilia A gene therapy? If I understand it correctly, they are both single gene defects and the same techniques should work.

From what I read about the mechanisms of the gene therapy, liver cells were ‘infected’ with a working F9 gene with their viral vector. The number of affected liver cells was proportional to the amount of viral vector given. So, if I understand this, once these liver cells die off the ability of the patient to produce Factor IX will go with them and they will need another round of therapy.

I would gladly have my liver treated every couple of years!

If any gene therapists are reading, I would gladly participate in an experimental trial. :P