遗传增强
病毒载体
载体(分子生物学)
基因治疗载体
计算生物学
医学
计算机科学
病毒学
基因
生物
遗传学
重组DNA
出处
期刊:Methods in molecular biology
日期:2022-01-01
卷期号:: 297-315
被引量:2
标识
DOI:10.1007/978-1-0716-2441-8_16
摘要
AbstractLentiviral vectors are among the most used vectors in gene therapy to treat pathologies of different origins, such as cancers, rare monogenic diseases or neurological disorders. This chapter provides an overview on lentiviral vector developments in terms of vector design and manufacture for gene therapy applications. The state of the art of vector production will be summarized face to the recent developments contributing to improve vector safety, efficacy and manufacturing robustness, focusing on human immunodeficiency virus 1 (HIV-1) based lentiviral vectors. Transient and stable production systems will be discussed highlighting recent advances in producer cell line development. Challenges in lentiviral vector development upstream and downstream will be addressed with a particular focus on the improvements undertaken to increase vector yields and production scalability.Key wordsLentivirusGene therapyManufactureSafetyPackaging cellsProductionBioreactionPurificationQuality
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