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  • Title: Retrovirus-mediated transfer of the human glucocerebrosidase gene to Gaucher fibroblasts.
    Author: Choudary PV, Barranger JA, Tsuji S, Mayor J, LaMarca ME, Cepko CL, Mulligan RC, Ginns EI.
    Journal: Mol Biol Med; 1986 Jun; 3(3):293-9. PubMed ID: 3736391.
    Abstract:
    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.
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