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机译:Effective cell and gene therapy in a murine model of Gaucher disease
Lund Univ, Inst Lab Med, Dept Mol Med & Gene Therapy, S-22184 Lund, Sweden;
Lund Univ, Lund Strateg Res Ctr Stem Cell Biol & Cell Therap, S-22184 Lund, Sweden;
Univ Lund Hosp, Dept Pathol, S-22100 Lund, SwedenSahlgrens Univ Hosp, Inst Clin Neurosci, S-43180 Molndal, SwedenNINDS, Dev & Metab Neurol Branch, NIH, Bethesda, MD 20892 USA;
HEMATOPOIETIC STEM-CELLS; HUMAN GLUCOCEREBROSIDASE GENE; BONE-MARROW TRANSPLANTATION; ENZYME REPLACEMENT THERAPY; ACID BETA-GLUCOSIDASE; IN-VIVO; N-BUTYLDEOXYNOJIRIMYCIN; RETROVIRAL VECTOR; MEDIATED TRANSFER; MOUSE MODEL;