I haven't followed the board in a long time, just wondering if anyone has heard this TED talk? I don't think HDSA is talking about it, sounds to me like the best hope for a full cure.


TED [www.ted.com] shared with TED for Windows Phone and Windows 8 Store apps.

Marsha I would love to hear your thoughts?



Edited 1 time(s). Last edit at 11/16/2015 12:15PM by MRO.

The whole technique is amazing. I remember my first HDSA convention when someone asked if it would be possible to edit out the extra CAG repeats and a researcher responded, "Wouldn't that be nice but we're taking about science fiction." Fifteen years later and science fiction is becoming a reality. I think that there is more research to be done before it can become a treatment but in the meantime we have gene silencing trials. I have a lot of hope for this technique, for gene silencing, and for stem cell restoration but I am getting frustrated with the pace.

I remember talking to someone on the old MGH chat room about 16 years. Back then I was all excited about the mouse models, the discovery of the HD protein aggregates and the possibility of delaying onset and progression with existing supplements and approved drugs while major treatments like gene silencing were being delayed. The person I was chatting with predicted a virtual cure in 15 to 20 years while I was more hopeful it would be sooner. He was right and I was wrong. I didn't realize how many delays occur in research. Getting funding for each stage, getting approval from the FDA, from IRBs, the gap in funding between an exciting potential treatment in the mice and all the work that has to be done before a clinical trial can take place, work that usually has to be funded and carried out before a big pharmaceutical company would be interested. I am like everyone else in the HD community in that we have learned things that most people never learn and things we never expected to need to know. I really am hopeful for the future, it is looking better and better, but I am just burned out with reading about HD research every day because I don't know where it is going or if it will get further funded and go anywhere. I am concentrating on the major trials now.