Correction of enzyme deficiency in metachromatic leukodystrophy fibroblasts by retroviral‐mediated transfer of the human arylsulphtase A gene

Long-term expression and transfer of arylsulfatase A into brain of arylsulfatase A-deficient mice transplanted with bone marrow expressing the arylsulfatase A cDNA from a retroviral vector

“…[18][19][20][21][22][23][24] Some of these vectors have been used to transduce fibroblasts of MLD patients and the virus-mediated hASA gene transfer led to a complete correction of the metabolic defect in these cells. 18 On the other hand it has been shown that overexpression of ASA has no adverse effects on the transduced cells.…”

Efficient and persistent expression of β‐glucuronidase gene in CD34+ cells from human umbilical cord blood by retroviral vector

“…Isolation of virus producing cell pMFG-HBG was cotransfected with pSV,Neo to Y-cre ecotropic packaging cells and selected by G418. About 50 clones were titered against NIH 3T3 cells as described (20). The amphotropic packaging cells, Y-crip, were infected using culture medium of highest titer ecotropic clone containing 8 yg/ml polybrene.…”

“…Infected Y-crip cells were plated-out at single cell/well in 96-well plates and expanded to 6-well plates. The medium from each 6-well plate was assayed for virus titer on NIH3T3 cells as described (20). The highest titer clone was used for the experiment.…”