Magnus Jørgen, Käsbach Jonas
RWTH Aachen University Institute of Biochemical Engineering Forckenbeckstr. 51 52074 Aachen Germany.
Med Genet. 2025 Jul 17;37(3):169-177. doi: 10.1515/medgen-2025-2024. eCollection 2025 Jul.
Adeno-associated virus (AAV) has become the leading viral vector for gene therapy. This review examines how different manufacturing methods, from mammalian to insect cell-based systems, influence AAV vector characteristics. Though each platform generates vectors with distinct molecular signatures affecting purity, safety and potency, clinical outcomes remain consistent across production platforms. Understanding the nuances of these platforms will still provide valuable insight for clinicians overseeing AAV-based therapy.
腺相关病毒(AAV)已成为基因治疗的主要病毒载体。本综述探讨了从哺乳动物细胞到昆虫细胞系统的不同生产方法如何影响AAV载体的特性。尽管每个平台产生的载体具有不同的分子特征,影响纯度、安全性和效力,但临床结果在各生产平台之间保持一致。了解这些平台的细微差别仍将为监督基于AAV的治疗的临床医生提供有价值的见解。