Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque

Ruth Seggewiss, Stefania Pittaluga, Rima L. Adler, F. Javier Guenaga, Cole Ferguson, Ingo H. Pilz, Byoung Ryu, Brian P. Sorrentino, W. Scott Young, Robert E. Donahue, Christof Von Kalle, Arthur W. Nienhuis, Cynthia E. Dunbar

Research output: Contribution to journalArticlepeer-review

109 Scopus citations

Abstract

We report, for the first time, a replication-defective retroviral vector-associated neoplasia in a nonhuman primate. Five years after transplantation with CD34+ cells transduced with a retroviral vector expressing enhanced green fluorescent protein (eGFP) and a drug-resistant variant of the dihydrofolate reductase gene (L22Y), a rhesus macaque developed a fatal myeloid sarcoma, a type of acute myeloid leukemia. Tumor cells contained 2 clonal vector insertions. One insertion was found in BCL2-A1, an antiapoptotic gene. This event suggests that currently available retroviral vectors may have long-term side effects, particularly in hematopoietic stem and progenitor cells.

Original languageEnglish
Pages (from-to)3865-3867
Number of pages3
JournalBlood
Volume107
Issue number10
DOIs
StatePublished - May 15 2006
Externally publishedYes

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