Creating Resistance to HIV Via CRISPR
Aug-04-2017 0 comments Cube Biosystems

Source: The Scientist, Anna Azvolinsky

Mice transplanted with human hematopoietic stem cells that have an HIV receptor gene, CCR5, disrupted by gene editing allows the animals to ward off HIV infection. 

A minor proportion of people harbor a homozygous mutation in CCR5, a gene that encodes a receptor found on immune cells, that thwarts HIV's attempts to get inside the cells. In an attempt to mimic this natural resistance, researchers mutated CCR5 in human fetal liver hematopoietic stem/progenitor cells (HSPCs) and showed that the cells could block HIV infection after transplantation into mice.

It should be noted that targeting CCR5 may not lead to a complete cure, as the virus is not elimated and has the potential to target different receptors, e.g. CXRC4. Yet, for now, CCR5 remains the best target.

Original Article: CRISPR/Cas9-mediated CCR5 ablation in human hematopoietic stem/progenitor cells confers HIV-1 resistance in vivo. Xu et al. Molecular Therapy.


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