Edited to clarify the difference between complete
reduction of huntingtin in the mouse experiments, as opposed to partial reduction by huntingtin - lowering drugs
Not exact matches
Huntingtin null neurons exhibit a significant
reduction in transcripts encoding proteins destined for the extracellular space, many
of which are components
of the extracellular matrix or involved in cellular adhesion, receptor binding and hormone activity.
We think the most exciting news in today's press release from Ionis is this: «In the Phase 1 / 2a study, dose - dependent
reductions of muntant
huntingtin were observed among patients treated with IONIS - HTTRx».
In addition, HDAC4
reduction delayed
huntingtin (Htt) aggregation in CNS tissue from both the R6 / 2 and HdhQ150 full length KI mouse model
of HD [1].