@peter_mantas Ah right, silence was the wrong word to use. Meant to say contract, at the DNA level. Very different from the current mHTT "silencing" approaches.
@peter_mantas The HTT gene being expressed is not inherently bad. It's about reaching a toxic repeat length and this compound shrinks repeat length back down to inherited or wild type levels. mHTT becomes normal HTT! Ofc, still not proven in humans or large animals...yet. ๐
@plainyogurt21 lot's of BBB tech out there similar to Denali, hopefully their success in hunter syndrome drives investment in the space, I have the perfect PoC enzyme to use, someone pls license or option it idc ๐