MULTIVALENT AAV VECTORS FOR HIV-1 GENE THERAPY

这个项目将检验这样的假设，即AAV载体能够 转导未分裂细胞并引导基因表达 独立的转录单位可以将抗HIV基因转导到 一种稳定的原始自我更新多能造血干细胞 时尚。编码多种抗HIV基因的多价AAV载体将被 测试它们对HIV-1的抵抗力。转导 效率、媒介整合和艾滋病毒抗药性将在#年进行评估 分化的后代。甲型肝炎病毒的长期转录状态 将对载体基因组进行评估，以确定所传递基因的命运 通过AAV载体。这项研究旨在提供以下信息 AAV载体基因转移的基础生物学研究进展 猪瘟基因治疗的抗病毒疗效、可行性和安全性 使用多价AAV载体的造血干细胞。

This project will test the hypothesis that AAV vectors which are capable of transducing non-dividing cells and directing gene expression from independent transcriptional units can transduce anti-HIV genes into primitive self-renewing pluripotent hematopoietic stem cells in a stable fashion. Multivalent AAV vectors encoding various anti-HIV genes will be tested for their ability to confer resistance to HIV-1. Transduction efficiencies, vector integration and HIV-resistance will be evaluated in the differentiated progeny. The long term transcriptional status of AAV vector genomes will be evaluated to determine the fate of genes delivered by AAV vectors. This study is designed to provide information regarding the basic biology of gene transfer with AAV vectors in addition to the anti-viral efficacy, feasibility and safety of gene therapy of hematopoietic stem cells using multivalent AAV vectors.