KSS's Latest, page-7

pga2003, the AAV-8 vector proved safe and efficacious in 10 patients, most whose lives have been tranformed and most do not now require life-saving blood transfusions. It has been a success but in this study but there was lower than expected transduction of hepatocytes which suggests that Mark Kay may be right. However, what is great is that it is still working years out and has no side effects.

For what it's worth, I personally don't think that TT-034 will make it. Too little too late. It is now merely a proof of concept study to prove ddRNAi is feasible for diseases that don't already have a cure. However, they are seeing evidence of the shRNA being produced in the first TT-034 patient which I wouldn't have expected if the AAV-8 is having difficulty transfecting hepatocytes, so if it cures patients at the higher doses, BLT will move very, very quickly.

Remember, this is about possible problems with the vector delivering the ddRNAi, not ddRNAi itself.