Liver-directed AAV gene therapy metabolically corrects AKU in Hgd deficient mice
Sien Lequeue, Brendan P. Norman, Gigly G. Del’haye…·Joery De Kock
This study demonstrates that liver-directed AAV gene therapy corrects metabolic defects in Hgd-deficient mice by restoring HGD expression and reducing HGA levels without hypertyrosinemia.