作者
Magalie Penaud-Budloo, Achille François, Nathalie Clément, Eduard Ayuso
发表日期
2018/3/16
来源
Molecular therapy Methods & clinical development
卷号
8
页码范围
166-180
出版商
Elsevier
简介
Recombinant adeno-associated viral (rAAV) vectors have been used in more than 150 clinical trials with a good safety profile and significant clinical benefit in many genetic diseases. In addition, due to their ability to infect non-dividing and dividing cells and to serve as efficient substrate for homologous recombination, rAAVs are being used as a tool for gene-editing approaches. However, manufacturing of these vectors at high quantities and fulfilling current good manufacturing practices (GMP) is still a challenge, and several technological platforms are competing for this niche. Herein, we will describe the most commonly used upstream methods to produce rAAVs, paying particular attention to the starting materials (input) used in each platform and which related impurities can be expected in final products (output). The most commonly found impurities in rAAV stocks include defective particles (i.e., AAV capsids that …
引用总数
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学术搜索中的文章
M Penaud-Budloo, A François, N Clément, E Ayuso - Molecular therapy Methods & clinical development, 2018