Project Alive - Gene Therapy
Health
The Gene Therapy
Well, I first became aware of hunter syndrome when my wife high in fu, started working for a laboratory at North Carolina that was specifically engaged in research in hunter syndrome. We began working with that group and trying to develop therapies specifically for that disease. Honda syndrome is a genetic disease with a single gene defect. The damage is very severe. However, we know what custody disease and the genes cause the disease being identified. So the difficult part is how do you get that gene in there? It's easy to clone the gene to grow it, but how do you get it into the patient? But we've developed it to the point now where we can, in the case of hunter, our plan is to do just a simple injection into the bloodstream, it appears that we can get it to go to many different essential organs and we can even get it to go to the brain. And that's been a real revolution for us.
The hanta community are our collaborators. Without them, this wouldn't be possible. We feel the inspiration from the kids and the parents, you know, driving us to continuously working on is no matter how difficult it is. In spite of all the obstacles, it's worthwhile to pursue this. There are enzyme replacement therapies that help, but they don't address the neurodegeneration and the neurodegeneration is what ultimately is robbing them of the lives that they really should have. It's working very well as a cure in the animal models. However, still back to the founding and a funding is the biggest hurdle when you're trying to move forward for clinical trial. Our goal and the goal of all our collaborators is to treat this patient with effective therapies and therefore we can further improve their qualified life and improve the quality of life after their families.