Drug Library Screening for the Identification of Ionophores That Correct the Mistrafficking Disorder Associated with Oxalosis Kidney Disease.

@article{Hou2017DrugLS,
  title={Drug Library Screening for the Identification of Ionophores That Correct the Mistrafficking Disorder Associated with Oxalosis Kidney Disease.},
  author={Shurong Hou and Franck Madoux and Louis D. Scampavia and Jo Ann Janovick and P. Michael Conn and Timothy P Spicer},
  journal={SLAS discovery : advancing life sciences R & D},
  year={2017},
  volume={22 7},
  pages={887-896}
}
Primary hyperoxaluria is the underlying cause of oxalosis and is a life-threatening autosomal recessive disease, for which treatment may require dialysis or dual liver-kidney transplantation. The most common primary hyperoxaluria type 1 (PH1) is caused by genetic mutations of a liver-specific enzyme alanine:glyoxylate aminotransferase (AGT), which results in the misrouting of AGT from the peroxisomes to the mitochondria. Pharmacoperones are small molecules with the ability to modify misfolded… CONTINUE READING