https://astx.com/wp-content/uploads/2019/11/logo_white.png 0 0 webadmin https://astx.com/wp-content/uploads/2019/11/logo_white.png webadmin2020-07-01 15:33:232020-07-01 15:33:232019 AD/PD: 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.