iBET, Instituto de Biologia Experimental e Tecnológica, Oeiras, Portugal.
Instituto de Tecnologia Química e Biológica António Xavier, Universidade Nova de Lisboa, Oeiras, Portugal.
Methods Mol Biol. 2022;2521:297-315. doi: 10.1007/978-1-0716-2441-8_16.
Lentiviral 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.
慢病毒载体是基因治疗中最常用的载体之一，可用于治疗不同起源的疾病，如癌症、罕见的单基因疾病或神经退行性疾病。本章概述了慢病毒载体在基因治疗应用方面的载体设计和制造方面的发展。将总结面对最近的发展，提高载体安全性、效力和制造稳健性的最新技术，重点介绍基于人类免疫缺陷病毒 1（HIV-1）的慢病毒载体。将讨论瞬时和稳定的生产系统，重点介绍在生产细胞系开发方面的最新进展。将解决慢病毒载体开发上下游的挑战，特别关注为提高载体产量和生产可扩展性而进行的改进。
