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非人类慢病毒载体的基因治疗应用。

Gene Therapy Applications of Non-Human Lentiviral Vectors.

机构信息

Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, University of Oxford, Oxford OX3 9DU, UK.

出版信息

Viruses. 2020 Sep 29;12(10):1106. doi: 10.3390/v12101106.

Abstract

Recent commercialization of lentiviral vector (LV)-based cell therapies and successful reports of clinical studies have demonstrated the untapped potential of LVs to treat diseases and benefit patients. LVs hold notable and inherent advantages over other gene transfer agents based on their ability to transduce non-dividing cells, permanently transform target cell genome, and allow stable, long-term transgene expression. LV systems based on non-human lentiviruses are attractive alternatives to conventional HIV-1-based LVs due to their lack of pathogenicity in humans. This article reviews non-human lentiviruses and highlights their unique characteristics regarding virology and molecular biology. The LV systems developed based on these lentiviruses, as well as their successes and shortcomings, are also discussed. As the field of gene therapy is advancing rapidly, the use of LVs uncovers further challenges and possibilities. Advances in virology and an improved understanding of lentiviral biology will aid in the creation of recombinant viral vector variants suitable for translational applications from a variety of lentiviruses.

摘要

最近,基于慢病毒载体(LV)的细胞治疗商业化和成功的临床研究报告表明,LV 在治疗疾病和造福患者方面具有尚未开发的潜力。与其他基因转移剂相比,LV 具有显著的内在优势,因为它们能够转导非分裂细胞、永久性地转化靶细胞基因组,并允许稳定、长期的转基因表达。基于非人类慢病毒的 LV 系统由于在人类中缺乏致病性,是传统 HIV-1 基于 LV 的有吸引力的替代品。本文综述了非人类慢病毒,并强调了它们在病毒学和分子生物学方面的独特特征。还讨论了基于这些慢病毒开发的 LV 系统及其成功和缺点。随着基因治疗领域的快速发展,LV 的使用揭示了进一步的挑战和可能性。病毒学的进步和对慢病毒生物学的更好理解将有助于创建适合各种慢病毒的翻译应用的重组病毒载体变体。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/60d9/7599719/b92ba17e7ab8/viruses-12-01106-g001.jpg

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