Mass spectrometry in gene therapy: Challenges and opportunities for AAV analysis

被引：10

作者：

Serrano, Mahalia A. C. ^{[1
]}

Furman, Ran ^{[1
]}

Chen, Guodong ^{[1
]}

Tao, Li ^{[1
]}

机构：

[1] Bristol Myers Squibb, Analyt Dev & Attribute Sci, Biol Dev Global Prod Dev & Supply, New Brunswick, NJ 10154 USA

来源：

DRUG DISCOVERY TODAY | 2023年 / 28卷 / 01期

关键词：

adeno-associated virus; gene therapy; mass spectrometry; native MS; charge detection MS; capsid viral protein; MOLECULAR-WEIGHT DETERMINATION; ADENOASSOCIATED VIRUS; VECTOR GENOME; CHO-CELLS; CHARGE; SEROTYPES; PARTICLE; QUANTIFICATION; EFFICIENT; PROTEINS;

D O I：

10.1016/j.drudis.2022.103442

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

The characterization of adeno-associated virus (AAV)-based gene therapy products represents significant challenges owing to their extremely large molecular sizes, structural complexity and heterogeneity, and limited sample amounts. Mass spectrometry (MS) is one of the key analytical tools that can overcome these challenges and serve as an important technique for the analysis of multiple attributes. In this review, the current methodologies and emerging trends in MS analysis of AAV gene therapy products are presented, highlighting their advantages and unique capabilities in addressing key issues encountered in intact AAV vector analysis, capsid viral protein characterization and impurity analysis.

引用

页码：1 / 10

页数：10

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