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MULTIVALENT AAV VECTORS FOR HIV-1 GENE THERAPY

MULTIVALENT AAV VECTORS FOR HIV-1 GENE THERAPY
用于 HIV-1 基因治疗的多价 AAV 载体
批准号：
2429509
负责人：
Saswati Chatterjee
金额：
$17.99万
依托单位：
CITY OF HOPE NATIONAL MEDICAL CENTER
依托单位国家：
美国
项目类别：
财政年份：
1996
资助国家：
美国
项目状态：
已结题
起止时间：
1996-07-01 至 1999-05-31
来源：
https://reporter.nih.gov/project-details/2429509
关键词：
DNA methylation HIV infections adeno associated virus group cell differentiation gene therapy genetic promoter element genetic transcription genetic transduction helper T lymphocyte hematopoietic stem cells human immunodeficiency virus 1 human subject macrophage monocyte nucleic acid chemical synthesis nucleic acid sequence posttranslational modifications tissue /cell culture transfection /expression vector virus replication

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中文摘要
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英文摘要
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.
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Genetic Modification of Human Hematopoietic Stem Cells with Pseudotyped rAAV
Genetic Modification of Human Hematopoietic Stem Cells with Pseudotyped rAAV
Genetic Modification of Human Hematopoietic Stem Cells with Pseudotyped rAAV
AAV TRANSDUCTION OF QUIESCENT HEMATOPOIETIC STEM CELLS
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