Identifies thymosin β4 as a therapeutic candidate for chronic granulomatous disease (CGD), a genetic NADPH oxidase deficiency with recurrent infections and hyperinflammation from defective autophagy. TB4 restored noncanonical autophagy through HIF-1α stabilization and DAPK1 activation, reducing excessive inflammasome activation in CGD cells. TB4 treatment improved phagocytic function and reduced pathological inflammation in human and murine CGD models. First application of TB4 to a genetic primary immunodeficiency—extending TB4's autophagy-promoting mechanism (identified in infection/repair contexts) to NADPH oxidase-deficient immune cells.
Renga, Giorgia; Oikonomou, Vasilis; Moretti, Silvia; Stincardini, Claudia; Bellet, Marina M; Pariano, Marilena; Bartoli, Andrea; Brancorsini, Stefano; Mosci, Paolo; Finocchi, Andrea; Rossi, Paolo; Costantini, Claudio; Garaci, Enrico; Goldstein, Allan L; Romani, Luigina