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1Molecular Microbiology & Immunology, University of Southern California Keck School of Medicine, Los Angeles, California, USA. pcannon@usc.edu
Current Opinion in HIV and AIDS
|January 19, 2011
Summary
Zinc finger nucleases (ZFNs) can disrupt the CCR5 gene, offering potential HIV resistance. This gene editing approach is being evaluated in clinical trials for T cells and hematopoietic stem cells (HSCs).
Area of Science:
- Immunology
- Gene Therapy
- Virology
Background:
- Individuals with CCR5Δ32 gene deletion are resistant to HIV-1.
- CCR5Δ32 gene editing in stem cells offers a potential HIV cure.
- This review focuses on disrupting CCR5 in autologous T cells and HSCs.
Purpose of the Study:
- To review recent developments in disrupting CCR5 expression in autologous T cells and HSCs.
- To explore gene editing strategies for HIV resistance.
- To assess the potential of CCR5 gene disruption for HIV therapy.
Main Methods:
- RNA interference for CCR5 suppression.
- Zinc finger nucleases (ZFNs) for CCR5 gene disruption.
- Nonhomologous end-joining pathway for gene editing.
- Humanized mouse models for preclinical studies.
Main Results:
- ZFNs can eliminate CCR5 expression in HIV-1 target cells.
- CCR5-targeted ZFNs demonstrated in vivo HIV-1 suppression in preclinical models.
- ZFNs are being evaluated in a phase I clinical trial for T cell therapy.
Conclusions:
- CCR5 gene knockout via ZFNs effectively suppresses HIV-1 replication in models.
- ZFN-mediated gene editing is a promising strategy for HIV therapy.
- Clinical trials are underway to evaluate ZFNs for T cell and HSC targeted therapies.
