Retrovirus-mediated transfer of the human glucocerebrosidase gene to Gaucher fibroblasts.
A recombinant retrovirus carrying complementary DNA containing the complete coding sequence for human glucocerebrosidase was constructed and used to infect mouse cells and human fibroblasts from a type 2 Gaucher patient. The cDNA stably integrated in the genome of the recipient cell. Infected cells made glucocerebrosidase-cross-reacting material, and the protein was glycosylated and contained the epitope(s) missing in the type 2 mutant glucocerebrosidase. This study demonstrates the feasibility of efficiently transferring the gene encoding glucocerebrosidase to Gaucher cells using retrovirus as vector.
D000818:Animals; D002478:Cells, Cultured; D003429:Cross Reactions; D004279:DNA, Viral / Q000235:genetics; D005347:Fibroblasts / Q000201:enzymology / Q000378:metabolism; D005776:Gaucher Disease / Q000201:enzymology / Q000235:genetics*; D005959:Glucosidases / Q000235:genetics*; D005962:Glucosylceramidase / Q000235:genetics* / Q000378:metabolism; D006801:Humans; D051379:Mice; D012190:Retroviridae / Q000235:genetics* / Q000502:physiology; D014162:Transfection; D014775:Virus Activation
D004279:DNA, Viral; D005959:Glucosidases; D005962:Glucosylceramidase