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. Author manuscript; available in PMC: 2017 Aug 1.
Published in final edited form as: Adv Drug Deliv Rev. 2016 Mar 21;103:174–186. doi: 10.1016/j.addr.2016.03.005

Fig 5. Schematic of potential RNAi therapy strategy for humans.

Fig 5

Two approaches can be used for human therapy. Either autologous CD4 T cells (purified from PBMC obtained by apheresis) or CD34+ HSPC (purified from bone marrow or blood after mobilization by GM-CSF treatment) can be in vitro modified to express shRNA (generally by transduction with lentivirus encoding CCR5 and antiviral shRNAs) and reinfused into the patients. Modified CD4 T cells provide a one-time source of HIV-1 resistant T cells. HSPC modification is expected to result in the continuous generation of HIV-1 resistant progeny T cells and macrophages.