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Lookup NU author(s): Dr Carolina Gandara de Souza,
Dr Elizabeth Stoll
This work is licensed under a Creative Commons Attribution 4.0 International License (CC BY 4.0).
© Carolina Gándara et al. 2018. Lentiviral vectors are used in laboratories around the world for in vivo and ex vivo delivery of gene therapies, and increasingly clinical investigation as well as preclinical applications. The third-generation lentiviral vector system has many advantages, including high packaging capacity, stable gene expression in both dividing and post-mitotic cells, and low immunogenicity in the recipient organism. Yet, the manufacture of these vectors is challenging, especially at high titers required for direct use in vivo, and further challenges are presented by the process of translating preclinical gene therapies toward manufacture of products for clinical investigation. The goals of this paper are to report the protocol for manufacturing high-titer third-generation lentivirus for preclinical testing and to provide detailed information on considerations for translating preclinical viral vector manufacture toward scaled-up platforms and processes in order to make gene therapies under Good Manufacturing Practice that are suitable for clinical trials.
Author(s): Gandara C, Affleck V, Stoll EA
Publication type: Article
Publication status: Published
Journal: Human Gene Therapy Methods
Print publication date: 01/02/2018
Online publication date: 24/01/2018
Acceptance date: 17/11/2017
Date deposited: 20/02/2018
ISSN (print): 1946-6536
ISSN (electronic): 1946-6544
Publisher: Mary Ann Liebert Inc.
PubMed id: PMC5806069
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