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Investigating drug delivery in Niemann-Pick type C

Olive was a D.Phil. student in the NIH-Oxford Scholar program. She studied the physiology of the blood-brain barrier (BBB) and how the BBB may facilitate disease progression. She is also interested in utilizing alternative drug delivery techniques, such as ultrasound (US) and microbubbles (MB) to disrupt the BBB and deliver drugs that normally cannot pass the BBB. In Prof. Platt’s lab, Olive investigated whether US+MB-based cyclodextrin delivery into Niemann-Pick type C mice by opening the BBB can potentially alleviate some of the disease phenotypes in these mice over the mid- to late-disease course.

Key points:

  • investigate in vivo the efficacy of cyclodextrin delivery via ultrasound-induced microbubble cavitation and disruption of the blood-brain barrier
  • understand whether the blood-brain barrier integrity is also compromised in Niemann-Pick type C disease course
  • identify any neurovascular differences between homeostatic and NPC-affected brains.

This work was supported by the NIH Oxford-Cambridge Scholars Program

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