Huntington’s disease: CAG RNAs induce DNA damage via NUDT16, drug candidate effective in vivo
05 Jun 2021
bởiNatalia Reoutova
An international team, including researchers from the Chinese University of Hong Kong (CUHK), demonstrated that neuronal DNA damage in Huntington’s disease (HD) is caused by untranslatable expanded CAG RNAs, which downregulate the expression of Nudix hydrolase 16 (NUDT16) gene. A bisamidinium compound, DB213, was subsequently shown to rescue CAG repeat RNA-induced synapse loss in a murine model of HD.