Table 1

Summary of results of clinical trials

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].

Marathe and Wooley Genetic Vaccines and Therapy 2007 5:5   doi:10.1186/1479-0556-5-5