Retroviral Vectors for Gene Transfer: Back from the Dead

Long-term follow-up of 43 HIV-infected patients who participated in gene-transfer trials using retroviral vectors showed persistent integration without adverse outcomes.

The 2009 case report of an HIV-infected man who was “functionally cured” after receiving a bone-marrow transplant from a CCR5-deficient donor (JW AIDS Clin Care Feb 13 2009) has raised hopes that target-cell modification may be a promising means of eradicating HIV. Because retroviruses are inherently capable of stable integration into the DNA of lymphocytes, they were long considered promising vectors for the transfer of genes into CD4 cells. Indeed, retroviral vectors were the mainstay of many early gene-transfer studies on the treatment of various diseases. However, in early trials using stem-cell targets, a disturbing number of patients developed leukemia after the retrovirus vector inserted near oncogenes. All ongoing gene-transfer tria…

Reviewing Author

Helmut Albrecht, MD

Disclosures

Grant / Research supportHealth Resources and Services Administration; Centers for Disease Control and Prevention

Leadership positions in professional societiesColumbia Medical Society; South Carolina Infectious Diseases Society

Helmut Albrecht, MD

Disclosures

Grant / Research supportHealth Resources and Services Administration; Centers for Disease Control and Prevention

Leadership positions in professional societiesColumbia Medical Society; South Carolina Infectious Diseases Society

Retroviral Vectors for Gene Transfer: Back from the Dead

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Reviewing Author