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Biostockclub

10/29/18 9:48 PM

#170258 RE: Pineapple1 #170255

Pineapple,


“Any thoughts on CRISPR to get the WT S1R into someone's cells and negate

variants?”


That’s great food for thought and certainly worth looking into, imo. Could be the way to help non or lessor respondents without WT derive benefit from our drug. Thank you for the thought provoking idea...Extra Effort!

I’ll look into that, as I’m sure others will, and others on the board may already have - if so, hope they will share whatever input gained as this could be a viable, valuable route to greater success in beating the disease with our drug response.
Excellent!


And, as long as you mentioned the amount of information in the articles (I really see why this was included in our slide, although almost overlookable), I have to add that I think it is no coincidence that the article cited contained this about the Sig 1 r:

“Sig-1R-lipid interactions are important in both oligodendrocyte (OL) differentiation and axon extensions. Sig-1Rs target galactosylceramide (GalCer)- and cholesterol-enriched lipid microdomains on the ER of OLs, and may thus modulate myelination by controlling the dynamics of the lipid transport to the myelin membrane [6].”

If that isn’t a dog whistle to BIIB, don’t know what is? (wink)

I’m sure Biogen has read the relevant research on myelination and oligodendrocytes, so this would have come as no surprise to them. Still, here we have it, once again, in black and white, scientifically published.

Things just keep looking better and better for this mechanism of action across the board for CNS.

Good stuff - thanks,
Bio