Liver-directed AAV gene therapy metabolically corrects AKU in Hgd deficient mice
Sien Lequeue, Brendan P. Norman, Gigly G. Del’haye, Jessie Neuckermans, Haaike Colemonts‐Vroninks, Juliette H. Hughes, Matthias Rombaut, Claes Paul, Anja Heymans, Yves Heremans, Gunter Leuckx, Anneleen Mortier, Lakshminarayan R Ranganath, James A. Gallagher, Tamara Vanhaecke, , 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.
bioRxiv · Jun 2, 2026 · PreprintGenetics