Does anyone know of any succesful gene therapy trials in humans?

Rb

To have many informations, you can see the link
http://www.ifpma.org/clinicaltrials.html

There have been several gene therapy clinical trials in humans so far... with mixed results. Early trials were poorly thought out in my opinion.

In retrospect, treating an inflammatory based disease like cystic fibrosis with a pro-inflammatory vector like adenovirus is not such a good idea. But even there, the unfortunate death of a young man with CF really had very little to do with the trial. He was a poor candidate for the trial and was pushed through the selection process against the clinician's directives. Further he was administered a larger than recommended dose of adenovirus vector. You would think that MDs of all people would know that more is not always better. Apparently not.

More recent trials that I know of include the replacement of the RPE65 gene in LCA (Leber's Congenital Amneurosis) patients. This blinding condition is rare enough to qualify as an "orphan" disease. Pre-clinical data from the canine model of LCA in Briard dogs shows remarkable restoration of vision using an AAV vector to deliver the RPE65 gene to just one eye. It will be very interesting to follow how this trial progresses later this year.

I have no idea which source I read this in, but I do remember reading in a scientific journal about successful gene therapy for alzheimers in some VERY initial stages! If I come across the article, I will post a link for everyone to read.

There are also successful results in the treatment of ADA (adenosine deaminase) deficiency. You can find several articles about it. Do you want me to send few of them?

There have been plenty of relatively successful human trials.

For CF and Duchenne MD the most promising methods look to be non-viral. For DMD work from Geroge Dickson's group in England.

There are anti-HIV trials going on in the US with lentiviruses expressing microRNA cassettes which look good too.

SCID patients in the UK and France benefited from the retrovirus based GT they received (although the French trial also led to leukaemias in some patients).