We next generated an HDAd vector that expresses the human TFEB cDNA under the control of a liver-specific phosphoenolpyruvate carboxykinase (PEPCK) promoter and a liver-specific enhancer (Brunetti-Pierri et al, 2005b; HDAd-TFEB; Supporting Information Fig S1) to investigate in vivo the therapeutic potential of TFEB gene transfer for treatment of the liver disease of AAT deficiency. The gene discussed is TFEB; the disease is alpha 1-antitrypsin deficiency.