Improving Lentiviral Transduction of CD34⁺ Hematopoietic Stem and Progenitor Cells.

Q1 Immunology and Microbiology Human Gene Therapy Methods Pub Date : 2018-04-01 DOI:10.1089/hgtb.2017.085

Ilona Hauber, Niklas Beschorner, Silke Schrödel, Jan Chemnitz, Nicolaus Kröger, Joachim Hauber, Christian Thirion

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引用次数: 24

Abstract

The delivery of therapeutic genes for treatment of inherited or infectious diseases frequently requires lentiviral transduction of CD34⁺ hematopoietic stem and progenitor cells (HSC). Optimized transduction protocols with a therapeutic goal aim to maximize the number of transduction-positive cells while limiting the vector copy number that reach each individual cell. Importantly, the transduced HSC should maintain their "stem-like" properties. Here, we analyzed LentiBOOST™ reagent, a membrane-sealing poloxamer, with respect to enhancing lentiviral transduction of CD34⁺ peripheral blood stem cells. We demonstrate that inclusion of LentiBOOST™ in a standard HSC transduction protocol yields high transduction efficiencies while preserving the ability of the transduced HSC to differentiate into various hematopoietic lineages. Thus, LentiBOOST™ reagent can significantly improve lentiviral CD34⁺ HSC transduction protocols with the potential to improve production of gene-modified cell products.

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改善CD34+造血干细胞和祖细胞的慢病毒转导。

用于治疗遗传性或感染性疾病的治疗基因的递送通常需要慢病毒转导CD34+造血干细胞和祖细胞(HSC)。优化转导方案的治疗目标是最大化转导阳性细胞的数量，同时限制到达每个细胞的载体拷贝数。重要的是，转导的HSC应保持其“茎样”特性。在这里，我们分析了LentiBOOST™试剂，一种膜密封poloxamer，在增强CD34+外周血干细胞慢病毒转导方面。我们证明，在标准的HSC转导方案中包含LentiBOOST™可产生高转导效率，同时保留转导的HSC分化成各种造血谱系的能力。因此，LentiBOOST™试剂可以显著改善慢病毒CD34+ HSC转导方案，有可能提高基因修饰细胞产品的生产。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Human Gene Therapy Methods BIOTECHNOLOGY & APPLIED MICROBIOLOGY-GENETICS & HEREDITY

CiteScore

5.80

自引率

0.00%

发文量

审稿时长

>12 weeks

期刊介绍： Human Gene Therapy is the premier, multidisciplinary journal covering all aspects of gene therapy. The Journal publishes in-depth coverage of DNA, RNA, and cell therapies by delivering the latest breakthroughs in research and technologies. Human Gene Therapy provides a central forum for scientific and clinical information, including ethical, legal, regulatory, social, and commercial issues, which enables the advancement and progress of therapeutic procedures leading to improved patient outcomes, and ultimately, to curing diseases. The Journal is divided into three parts. Human Gene Therapy, the flagship, is published 12 times per year. HGT Methods, a bimonthly journal, focuses on the applications of gene therapy to product testing and development. HGT Clinical Development, a quarterly journal, serves as a venue for publishing data relevant to the regulatory review and commercial development of cell and gene therapy products.