Publications by authors named "Evan Lopez"
Gene therapy for HIV-1 infection is a promising alternative to lifelong combination antiviral drug treatment. Chemokine receptor 5 (CCR5) is the coreceptor required for R5-tropic HIV-1 infection of human cells. Deletion of CCR5 renders cells resistant to R5-tropic HIV-1 infection, and the potential for cure has been shown through allogeneic stem cell transplantation with naturally occurring homozygous deletion of CCR5 in donor hematopoietic stem/progenitor cells (HSPC).
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- Research is focused on developing genetic strategies to block the CCR5 co-receptor, which is essential for HIV-1 infection, as a therapy for HIV.
- Hematopoietic stem/precursor cells (HSPC) can be modified to lack CCR5 using zinc finger nucleases (ZFNs), allowing for the creation of HIV-resistant CD4(+) T cells after transplantation.
- The effectiveness and safety of these gene therapies can be tested by transplanting ZFN-treated HSPC into immunodeficient mice, which serve as a useful model for studying their potential against HIV.
The HIV-1 coreceptor CCR5 is a validated target for HIV/AIDS therapy. The apparent elimination of HIV-1 in a patient treated with an allogeneic stem cell transplant homozygous for a naturally occurring CCR5 deletion mutation (CCR5(Δ32/Δ32)) supports the concept that a single dose of HIV-resistant hematopoietic stem cells can provide disease protection. Given the low frequency of naturally occurring CCR5(Δ32/Δ32) donors, we reasoned that engineered autologous CD34(+) hematopoietic stem/progenitor cells (HSPCs) could be used for AIDS therapy.
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