WIKICOMMONS, AJC1A 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.
“These are expected observations that support prior data that targeting CCR5 can impede HIV replication and spread,” says neuroscientist Kamel Khalili who studies HIV infection at Temple University’s Lewis Katz School of Medicine in Philadelphia and who was not involved in the study. “CRISPR may be more convenient for gene editing than the prior gene editing tools used.”
The study is not the first to use gene editing to interrupt the CCR5 gene in human hematopoietic stem/progenitor cells. In a study published in 2010, Paula Cannon and her colleagues used zinc-finger nucleases to disrupt the gene and showed that the edited cells, when engrafted into mice, could clonally expand and retain the CCR5 deletion. This previous work has led to an on-going clinical trial to ...
