Re:Trial shows Huntington's drug could slow progress of disease
by ghkbrew on Monday December 11, 2017 @12:07PM
(#15898362)
Impressive. An important point here seems to be that these ASO's have a chemically modified backbone which prevents degradation by exonucleases. But the bases are un-modified allowing complimentary pairing and activation of RNAse H activity. Even so, I'm surprised they're able to cross the cell membrane in high enough quantities to have therapeutic effects.
Apparently, these techniques have been around since the mid-nineties or so. TIL...
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