Hi Rick,
"Gene expression was not demonstrated"
They're pretty vague about that(It makes me wonder why?), my guess is that they tested for it, and didn't detect any expression...so either they didn't test for it, the DNA got kicked out of the cells were expressing it, the expressing cells died (what is the lifespan for normal lung tissue?) or were destroyed before the testing time point, the gene was silenced, or ? I also wonder what time pts they used to test for expression(if they tested for it), day 30, 60, 90? Also was there an immune response to the expressed gene? Too many unanswered questions...til the data is released...who knows. But I think in Avigen's AAV trials, they were expecting booster shots every 2-3 yrs, not 30 days.
Message 16670812
BWDIK?
Best of luck,
SS |