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HIV Gene Therapy Safe and Effective

Has modest efficacy for reducing viral load and raising lymphocyte counts

MONDAY, Feb. 16 (HealthDay News) -- Gene therapy using a ribozyme that targets HIV RNA is safe and has modest efficacy in reducing viral load and raising CD4+ T cell counts, according to study findings published online Feb. 15 in Nature Medicine.

As part of a phase 2 clinical trial, Ronald T. Mitsuyasu, M.D., from the University of California Los Angeles, and colleagues randomly assigned 74 HIV-1-infected patients to a single infusion of a placebo or gene therapy with a tat-vpr-specific anti-HIV ribozyme (OZ1) delivered via autologous CD34+ hematopoietic progenitor cells.

The investigators found that mean plasma viral loads were lower in the OZ1 group after 47 and 48 weeks. Although this did not reach statistical significance, a higher percentage of patients in the OZ1 group had a viral load of less than 4 log10 copies per milliliter at this time point, the researchers report. In addition, the time-weighted areas under the curve were significantly lower in the OZ1 group at 40 to 48 weeks and at 40 to 100 weeks. The number of CD4+ lymphocytes was also higher in the OZ1 group throughout the 100 weeks, the report indicates. There were no serious adverse events associated with gene therapy, the authors note.

"This study indicates that cell-delivered gene transfer is safe and biologically active in individuals with HIV and can be developed as a conventional therapeutic product," Mitsuyasu and colleagues conclude.

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