Would a trojan horse approach to get antisense oligos (ASO) into the brain, via intravenous infusion of xB3/Mtfp-ASO, be as clinically effective as an intrathecal injection?
"The three-month randomized, placebo-controlled, dose escalation Phase 1/2a clinical study will evaluate the safety and activity of IONIS-MAPTRx in approximately 44 patients with mild AD. In the study, IONIS-MAPTRx is administered as a once-monthly intrathecal injection directly into the cerebral spinal fluid, similar to the way SPINRAZA(r) (nusinersen) is administered."
http://ir.ionispharma.com/news-releases/news-release-details/ionis-pharmaceuticals-initiates-clinical-study-ionis-mapt-rx