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Practical Application of Rodent Transporter Knockout Models to Assess Brain Penetration in Drug Discovery.

Elin EnebergChristopher R JonesThomas JensenKristine LangthalerChristoffer Bundgaard
Published in: Drug metabolism and bioanalysis letters (2022)
While in vitro assays showed compound X to be a substrate for human BCRP and not P-gp, in vivo studies indicated a synergistic effect between rodent efflux transporters. However, this only accounted for ~50% of restricted BBB-transport, suggesting involvement of other efflux transporters. Considering Kp, uu as a key criterion for assessing the technical quality of CNS candidates before progression into clinical development, it is important to identify relevant screening assays for a better understanding of low Kp, uu and brain distribution in pre-clinical models for translation to humans.
Keyphrases
  • drug discovery
  • white matter
  • blood brain barrier
  • resting state
  • endothelial cells
  • high throughput
  • functional connectivity
  • multiple sclerosis
  • induced pluripotent stem cells