Year 2

In the second year of the CIRM ETR2-01771 project entitled “Development of RNA-based approaches to stem cell gene therapy for HIV” we have made significant progress in the development of a robust animal model of human hematopoietic stem and progenitor cell (HSPC) engraftment, completed the cloning and in vitro testing of the candidate second generation anti-HIV lentiviral vectors and begun testing HIV infectivity of humanized NSG mice. Additionally, we have initiated transplantation of NSG mice with gene modified HSPC to evaluate the level of gene modified CD4+ T-cells and monocytes in vivo. This work will culminate in HIV challenge assays in the final year of the grant and selection of the second generation candidate therapeutic to be used in our clinical development program.