T cell-based HIV gene therapy safe over long term

May 7, 2012
T cell-Based HIV gene therapy safe over long term

(HealthDay) -- T cell-based gene therapy for HIV seems safe, with no evidence of vector-induced cell immortalization more than a decade after treatment, according to a study published in the May 2 issue of Science Translational Medicine.

John Scholler, from the University of Pennsylvania Perelman School of Medicine in Philadelphia, and colleagues performed a follow-up of HIV-infected patients who had received consisting of engineered with linked to the CD3ζ signaling chain as part of three clinical trials at least eleven years earlier.

The researchers found that the engineered T cells were still detectable in 98 percent of samples, with no evidence of vector-induced immortalization. There was no evidence of persistent clonal expansion or enrichment for integration sites near genes implicated in transformation or growth control. The modified cells were stably engrafted, with a half-life of at least 16 years, and remained functional.

"Our results emphasize the safety of T cells modified by retroviral gene transfer in clinical application, as measured in >500 patient-years of follow-up," Scholler and colleagues conclude. "Thus, previous safety issues with integrating viral vectors are hematopoietic stem cell or transgene intrinsic, and not a general feature of retroviral vectors."

One author is employed by Celgene; another author disclosed working as an advisor and clinical investigator for biopharmaceutical companies.

Explore further: The promise of stem cell-based gene therapy

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