Title : Liver-directed AAV gene therapy normalizes disease symptoms and provides cross-correction in a mouse model of lysosomal acid lipase deficiency - Lam_2024_Mol.Ther__
Title : Therapeutic efficacy of rscAAVrh74.miniCMV.LIPA gene therapy in a mouse model of lysosomal acid lipase deficiency - Lam_2022_Mol.Ther.Methods.Clin.Dev_26_413