Defining the mechanism of action of 4-phenylbutyrate to develop a small-molecule-based therapy for Alzheimer's disease.

@article{CuadradoTejedor2011DefiningTM,
  title={Defining the mechanism of action of 4-phenylbutyrate to develop a small-molecule-based therapy for Alzheimer's disease.},
  author={Mar Cuadrado-Tejedor and Ana Garc{\'i}a-Osta and Ana L Ricobaraza and Julen Oyarz{\'a}bal and Roberto Jorge da Silva Franco},
  journal={Current medicinal chemistry},
  year={2011},
  volume={18 36},
  pages={
          5545-53
        }
}
4-phenylbutyrate (PBA) is a small molecule that restores cognitive deficits in animal models of Alzheimer's disease (AD). Although the molecular basis of the cognitive benefits of PBA remains unknown, a multi-modal/multi-target mechanism has been proposed. Putative targets of this drug are different from those of drugs that are now used in clinical trials. As PBA is already administered to patients with congenital defects affecting enzymes in the urea cycle, it can be rapidly tested in AD… CONTINUE READING

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