Retrovirus-mediated transfer of the human glucocerebrosidase gene to Gaucher fibroblasts.

Prabhakara V Choudary, J. A. Barranger, S. Tsuji, J. Mayor, M. E. LaMarca, C. L. Cepko, R. C. Mulligan, E. I. Ginns

Research output: Contribution to journalArticlepeer-review

30 Scopus citations


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.

Original languageEnglish (US)
Pages (from-to)293-299
Number of pages7
JournalMolecular biology & medicine
Issue number3
StatePublished - Jun 1986
Externally publishedYes

ASJC Scopus subject areas

  • Molecular Biology
  • Genetics(clinical)


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