| 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__ |
| Author(s) : Lam P , Zygmunt DA , Ashbrook A , Yan C , Du H , Martin PT |
| Ref : Mol Ther , : , 2024 |
| Abstract : |
| PubMedSearch : Lam_2024_Mol.Ther__ |
| PubMedID: 39489913 |
| Gene_locus related to this paper: mouse-1llip |
| 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 |
| Author(s) : Lam P , Ashbrook A , Zygmunt DA , Yan C , Du H , Martin PT |
| Ref : Mol Ther Methods Clin Dev , 26 :413 , 2022 |
| Abstract : |
| PubMedSearch : Lam_2022_Mol.Ther.Methods.Clin.Dev_26_413 |
| PubMedID: 36092360 |
| Gene_locus related to this paper: mouse-1llip , human-LIPA |