The rAAV2 serotype 5 (rAAV2/5) vector, containing human cDNA of the housekeeping PBGD isoform, stably sustained hepatic PBGD transgene expression and prevented the accumulation of porphyrins during the acute attacks in not only male but also female AIP mice, which are usually less susceptible to rAAV-mediated gene transfer [93]. Here, HMBS is linked to autoimmune pancreatitis.