Sangamo BioSciences' (SGMO) first clinical study, a phase 1 trial conducted on six HIV-positive patients over 12 weeks, demonstrates that the T-cell genome can be safely engineered to mimic a naturally occurring mutation that provides resistance to HIV infection. This could potentially be a way to control HIV infection without the use of drugs.
In the study, T-cells from the subjects were isolated and edited with ZFNs to make them resistant to the HIV virus by knocking out the CCR5 gene that encodes a coreceptor required for infection. The altered cells, SB-728-T, were returned to the subjects via autologous infusion.
The ZFN-modified cells showed preferential survival over unmodified cells and were able to persist and circulate throughout the body to key reservoirs of HIV infection such as gut-associated lymphoid tissue.
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