Stable reduction of CCR5 by RNAi through hematopoietic stem cell transplant in non-human primates

通过非人类灵长类动物造血干细胞移植,利用 RNAi 稳定降低 CCR5

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作者:Dong Sung An, Robert E Donahue, Masakazu Kamata, Betty Poon, Mark Metzger, Si-Hua Mao, Aylin Bonifacino, Allen E Krouse, Jean-Luc Darlix, David Baltimore, F Xiao-Feng Qin, Irvin S Y Chen

Abstract

RNAi is a powerful method for suppressing gene expression that has tremendous potential for therapeutic applications. However, because endogenous RNAi plays a role in normal cellular functions, delivery and expression of siRNAs must be balanced with safety. Here we report successful stable expression in primates of siRNAs directed to chemokine (c-c motif) receptor 5 (CCR5) introduced through CD34+ hematopoietic stem/progenitor cell transplant. After hematopoietic reconstitution, to date 14 months after transplant, we observe stably marked lymphocytes expressing siRNAs and consistent down-regulation of chemokine (c-c motif) receptor 5 expression. The marked cells are less susceptible to simian immunodeficiency virus infection ex vivo. These studies provide a successful demonstration that siRNAs can be used together with hematopoietic stem cell transplant to stably modulate gene expression in primates and potentially treat blood diseases such as HIV-1.

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