2019 AD/PD: Discovering small-molecule modulators of autophagy for neurodegeneration

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Discovering Small-Molecule Modulators of Autophagy for Neurodegeneration


  • Autophagy is a major protein degradation pathway with proven roles in protecting neurons against accumulation of aggregation-prone proteins and obsolete mitochondria. Genetics of human-disease and mouse-knockout studies highlight the connection between autophagy dysfunction and neurodegeneration, and therefore finding a way to augment autophagic-flux seems a promising therapeutic strategy (Rubinsztein DC, Bento CF & Deretic V. Journal of Experimental Medicine 2015; Bento CF et al. Annual Review of Biochemistry 2016).
  • Fragment-based drug discovery (FBDD) has the potential to deliver potent, selective and CNS-penetrant small-molecules capable of inducing autophagy in the brain. For that purpose, the use of cell-based assays that accurately monitor the ability of small-molecules to modulate autophagy in a high-throughput- and unbiased-manner is instrumental.