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Harnessing HIV for therapy, basic research and biotechnology

机译:利用艾滋病毒进行治疗,基础研究和生物技术

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First described about a decade ago, lentiviral vectors ('lentivectors') have emerged as potent and versatile tools of gene transfer for basic and applied research and offer exciting perspectives for the field of gene therapy. In the clinic, HIV-based vectors are showing particular promise for delivering therapeutic genes to hematopoietic stem cells (HSCs) and terminally differentiated targets in the central nervous system (CNS). Their flexible design facilitates the accommodation of sophisticated elements of control for the precise tuning of transgene expression. The delivery of small interfering RNAs (siRNAs) and genomic or cDNA libraries and the creation of transgenic animals are the most recent and exciting applications of HIV-based vectors that will help to tackle fundamental issues across wide areas of biology.
机译:慢病毒载体(“ lentivectors”)首次描述于大约十年前,已成为基础和应用研究的强大而通用的基因转移工具,并为基因治疗领域提供了令人兴奋的观点。在临床中,基于HIV的载体显示出将治疗基因递送至造血干细胞(HSC)和中枢神经系统(CNS)终末分化靶标的特殊前景。其灵活的设计有助于适应复杂的控制元件,以精确调节转基因表达。小型干扰RNA(siRNA)和基因组或cDNA文库的传递以及转基因动物的创造是基于HIV的载体的最新和令人兴奋的应用,它将帮助解决广泛的生物学领域的基本问题。

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