We have had hopes for HD treatments dashed many times before. It’s hard to not be cynical, but this one seems like it could be the real thing.<p>Godspeed to everybody working on this...with an emphasis on the “speed” part.
Does anyone have a link to a more technical description of this therapy? From what I've found it's a small anti-sense RNA targeting the mutant huntingtin gene. But I don't see a description of a delivery vehicle, or if there even is one. The drug seems to have pretty big implications if it works, but I'll be frankly amazed if all they had to do was inject the bare nucleic acid into the CSF.