Inhibition of USP2 suppresses pathological pexophagy and rescues PEX1-deficient peroxisome biogenesis disorder
Dear Editor , Peroxisome biogenesis disorders (PBDs) are ultrarare, life-limiting inherited diseases in which functional peroxisomes are pathologically lost, driving progressive multisystem failure dominated by neurological involvement. 1 Clinically, PBDs are encompassed within the Zellweger spectrum disorders (ZSDs)