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Table 1 Summary of results of clinical trials

From: Is gene therapy a good therapeutic approach for HIV-positive patients?

Target cells Vector Transgene Anti-HIV method Results
CD8+ Retrovirus HyTk Introduction of suicide gene CTL response cleared modified cells [16].
CD4+ Gold-particle-mediated Rev M10 Transdominant negative protein Detected Rev M10 until 2 months post infusion, preferential survival [36].
CD4+ Retrovirus Rev M10 Transdominant negative protein Detected Rev M10 until 6 months post infusion, preferential survival [37].
CD4+ Retrovirus TdRev and/or anti-sense TAR Transdominant negative proteins and anti-sense RNA Anti-HIV genes consistently detected for >100 weeks in six of six patients. Preferential survival of transduced cells during a period of high viral load in one patient [47].
CD34+ Retrovirus TdRev Transdominant negative protein One patient died due to relapse to Hodgkin's disease. In second patient, detected vector in the progeny for >3 years, remission of leukemia and good viral load control achieved by administering HAART that cannot be attributed to gene therapy [38,39].
CD34+ Retrovirus huM10 Transdominant negative protein huM10 could be detected in peripheral blood mononuclear cells (PBMC) for 1–3 months and then dropped to at or below the limit of detection over a two year follow-up period. Preferential survival of transduced cells during a period of high viral load in one patient [40].
CD4+ Retrovirus CD4ζ Chimeric receptor Decrease of greater than 0.5 log mean in rectal tissue-associated HIV RNA for at least 14 days, detected CD4ζ in 1–3% of PBMCs at 8 weeks [42]
CD4+ Retrovirus CD4ζ Chimeric receptor Good expression of CD4ζ for at least 24 weeks in all patients; no difference between control and study group [43].
CD4+ and CD8+ Retrovirus CD4ζ Chimeric receptor In 11 of 12 patients who received higher doses of modified CD8+ cells (109 or 1010), CD4ζ could be detected post-infusion for at least 15–40 weeks when they received additional infusions of modified cells. The group receiving IL-2 along with modified CD8+ cells showed a higher persistence of CD4ζ as compared to the group receiving no IL-2. In patients who received modified CD8+ and CD4+ cells, the cells were detected in the peripheral blood for at least 1 year post-infusion [41].
CD34+ Retrovirus (RRE) decoy RNA decoy RRE-decoy-containing leukocytes could be isolated from peripheral blood even 1 year post-infusion but the numbers were extremely low [44].
CD4+ Retrovirus RRz2 Ribozyme Over a 4 year period, PBMCs containing both RRz2 and LNL6 were consistently detected [46].
CD34+ Retrovirus tat/vpr ribozyme Ribozyme Vector was detected in naïve T cells for >3 years; no correlation between changes in viremia or CD4+ T cell counts with vector expression or its detection in any cell type [45].