Molecular BiologyCommunications MedicinePeer-reviewed
Rescue of lysosomal acid lipase deficiency in mice by rAAV8 liver gene transfer.
Marine Laurent, Rim Harb, Christine Jenny…·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.
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