Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transfer.
Marine Laurent, Rim Harb, Christine Jenny, Julie Oustelandt, Simon Jimenez, Jérémie Cosette, Francesca Landini, Aristide Ferrante, Guillaume Corre, Nemanja Vujić, Cláudia Piccoli, Anaïs Brassier, Laetitia van Wittenberghe, Giuseppe Ronzitti, Dagmar Kratky, , Mario Amendola
The paper develops a liver-targeted gene therapy using recombinant AAV8 to cure Lysosomal acid lipase deficiency by correcting lipid accumulation and normalizing gene expression and mitochondrial function in a mouse model.
Communications Medicine · Apr 11, 2025Peer-reviewedMolecular Biology