Gene and Cellular Therapy Core

高效抗逆转录病毒疗法的出现导致机会性感染的发生率下降， 感染和延长HIV-1感染者的生存期。病毒入侵的障碍包括 持续存在感染的静止池、耐药性、毒性和缺乏治疗 坚持。基因疗法的目标是用细胞替代HIV-1感染的细胞库 这些病毒经过基因改造可以抵抗HIV-1的复制。与传统药物不同， 创建一个“单次给药”试剂。然而，成功的基因治疗策略需要高效的基因治疗。 递送到造血干细胞中，以及抗HIV基因在造血干细胞中的存在和表达。 如果不是在个体的一生中持续多年的分化后代。因此，目前的这些限制 必须进行调查。更好地理解基因治疗技术的局限性可以导致 战略，以克服这些限制，以及设计潜在的临床情况下，测试他们。作为 干细胞基因治疗策略是抗逆转录病毒疗法的可能替代或补充， 科学界，特别是卫生政策界和广大公众的利益。 基因和细胞治疗核心（核心G）的总体目标是提供科学和技术 支持需要使用纯化CD 34+造血干细胞的基础实验室和临床试验项目 干细胞用于HIV/AIDS的基因治疗、造血和HIV的发病机制。

The advent of highly active antiretroviral therapy has resulted in a decline in the incidence of opportunistic infections and prolonged survival in HIV-1 infected individuals. Obstacles towards virus erradication include the continued presence of an infected quiescent pool, drug resistance, toxicities, and lack of treatment adherence. The goal of genetic therapies is the replacement of the HIV-1-infected cell reservoir with cells that have been genetically engineered to resist HIV-1 replication. Unlike conventional drugs, it is possible to create a "single administration" reagent. However, successful gene therapy strategies require efficient gene delivery into hematopoietic stem cells and the presence and expression of the anti HIV genes in differentiated progenies that persist for years if not for the life of the individual. Thus, these current limitations must be investigated. A better understanding of the limitations of gene therapy techniques can lead to strategies to overcome such limitations as well as design potential clinical scenarios in which to test them. As possible alternatives or complements to antiretroviral therapy, stem cell gene therapy strategies are of interest to the scientific community, to the health policy community in particular and to the public at large. The overall goal of the Gene and Cellular Therapy Core (Core G) is to provide scientific and technical support for basic laboratory and clinical trial projects that require the use of purified CD34+ hematopoietic stem cells for HIV/AIDS gene therapy, hematopoiesis and pathogenesis of HIV.