Gene and Cellular Therapy Core

高效抗逆转录病毒疗法的出现导致机会性疾病的发生率下降。 艾滋病毒-1感染者的感染和延长存活时间。根除病毒的障碍包括 感染静止池的持续存在、耐药性、毒性和缺乏治疗 坚持不懈。基因疗法的目标是用细胞取代HIV-1感染的细胞库 这些基因工程已经被用来抵抗HIV-1复制。与传统药物不同，它有可能 创造一种“单一给药”试剂。然而，成功的基因治疗策略需要有效的基因。 造血干细胞转导及抗HIV基因的存在和表达 如果不是在个体的一生中，也能存活数年的分化后代。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 克服这些限制的策略，以及设计潜在的临床场景来测试它们。AS 作为抗逆转录病毒治疗的可能替代或补充，干细胞基因治疗策略有 科学界，特别是卫生政策界和广大公众的兴趣。 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 支持需要使用纯化的CD34+造血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.