I hope this potential proves to be right…
“Gene therapy approaches that involve the genetic modification of human haematopoietic stem cells have the potental to engineer HIV control by introducing cells resistant to HIV infection, the 20th Conference on Retroviruses and Opportunistic Infections (CROI 2013) heard last week.
The proof of concept for this approach to HIV control in humans comes from the case of the ‘Berlin patient’, who received a bone marrow transplant from a donor with natural resistance to HIV infection. The donor was homozygous for the CCR5 delta 32 mutation, meaning that cells potentially vulnerable to HIV infection would never display the CCR5 receptor necessary for HIV to gain entry to that cell. As a result of complete ablation of the recipient’s own stem cells by chemotherapy, his CD4 cells were replaced by cells derived from the donor’s CCR5-lacking population. Over three years after the procedure the recipient remains HIV-free without antiretroviral treatment, and has been described as ‘functionally cured’ by physicians.
However, the chance of reproducing this outcome using transfer of donor cells is very low due to the shortage of potential donors who are both HLA-compatible (essential for avoidance of graft-versus-host disease) and CCR5-delta32 homozygous. In any case, the essential elements that contributed to this functional cure are still not fully understood.
The introduction of modified genes into stem cells harvested from a person’s bone marrow will be a necessarily individualized treatment, but the costs of gene therapy are likely to come down in the future, and if experimental approaches prove successful in controlling HIV without antiretroviral drugs, gene therapies may deliver a cost-effective form of treatment in the future…”
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