Bone marrow gene therapy remains an attractive option for treating chronic\nimmunological diseases, including acquired immunodeficiency syndrome (AIDS) caused\nby human immunodeficiency virus (HIV). This technology combines the differentiation\nand expansion capacity of hematopoietic stem cells (HSCs) with long-term expression of\ntherapeutic transgenes using integrating vectors. In this review we summarize the potential\nof bone marrow gene therapy for the treatment of HIV/AIDS. A broad range of antiviral\nstrategies are discussed, with a particular focus on RNA-based therapies. The idea is\nto develop a durable gene therapy that lasts the life span of the infected individual, thus\ncontrasting with daily drug regimens to suppress the virus. Different approaches have been\nproposed to target either the virus or cellular genes encoding co-factors that support virus\nreplication. Some of these therapies have been tested in clinical trials, providing proof of\nprinciple that gene therapy is a safe option for treating HIV/AIDS. In this review several\ntopics are discussed, ranging from the selection of the antiviral molecule and the viral\ntarget to the optimal vector system for gene delivery and the setup of appropriate preclinical\ntest systems. The molecular mechanisms used to formulate a cure for HIV infection are\ndescribed, including the latest antiviral strategies and their therapeutic applications. Finally,\na potent combination of anti-HIV genes based on our own research program is described.
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