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Gene-Based Stem Cell Therapy Knocks Down HIV Receptor Expression

UCLA researchers have reported successfully removing CCR5 from human cells in a humanized mouse model. CCR5 is a cell receptor to which HIV-1 binds for infection, but which the human body apparently does not need. Individuals who naturally lack the CCR5 receptor have been found to be essentially resistant to HIV. In the humanized mouse model, the researchers transplanted a specific short hairpin RNA (shRNA), targeted against the CCR5 gene, into human blood stem cells, in order to inhibit the expression of CCR5 in the human immune cells arising from the stem cells. According to the authors, the positive results provide evidence that this strategy may be an effective way to treat HIV-infected individuals, i.e., by prompting the potent long-term and stable reduction of CCR5 in systemic lymphoid organs. The results of the UCLA work were published in the February 25 issue of Blood. [Press release] [Blood abstract]