This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Objective: Many synthetic genes have been developed which can inhibit infection or replication of HIV-1 using a gene transfer/gene therapy approach, including genes encoding antisense RNA, ribozymes, dominant-negative mutant HIV-1 genes, RNA decoys, and intracellular antibodies against HIV-1 proteins or essential cellular co-factors. The limitation in targeting mature lymphocytes for gene therapy, the predominant host cell for HIV-1 infection, is the limited life-span of these mature cell populations. In these studies, we are assessing whether conditioning therapy (busulfan) is necessary for transplant of autologous transduced hematopoietic stem cells. Busulfan produces a specific loss of early stem cells and has been used as a stem cell cytotoxic conditioning agent prior to bone marrow transplantation in humans.
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