Folate-conjugated rapamycin slows progression of polycystic kidney disease.

@article{Shillingford2012FolateconjugatedRS,
  title={Folate-conjugated rapamycin slows progression of polycystic kidney disease.},
  author={Jonathan M. Shillingford and Christopher Paul Leamon and Iontcho Radoslavov Vlahov and Thomas Weimbs},
  journal={Journal of the American Society of Nephrology : JASN},
  year={2012},
  volume={23 10},
  pages={
          1674-81
        }
}
Activation of the mammalian target of rapamycin (mTOR) signaling pathway is aberrant in autosomal-dominant polycystic kidney disease (ADPKD). The mTOR inhibitors, such as rapamycin, ameliorate PKD in rodent models, but clinical trials have not shown benefit, possibly as a result of low tissue concentrations of rapamycin at clinically tolerable doses. To overcome this limitation, we synthesized a folate-conjugated form of rapamycin (FC-rapa) that is taken up by folate receptor-mediated… CONTINUE READING
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