A clinician's guide to AAV production - How manufacturing platforms shape vector properties.

IF 1.4 4区生物学

Medizinische Genetik Pub Date : 2025-07-17 eCollection Date: 2025-07-01 DOI:10.1515/medgen-2025-2024

Jørgen Magnus, Jonas Käsbach

引用次数: 0

Abstract

Adeno-associated virus (AAV) has become the leading viral vector for in vivo 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.

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AAV生产的临床医生指南-制造平台如何塑造矢量属性。

腺相关病毒（AAV）已成为体内基因治疗的主要病毒载体。本文综述了不同的制造方法，从哺乳动物到昆虫细胞为基础的系统，如何影响AAV载体特性。尽管每个平台产生的载体具有不同的分子特征，影响其纯度、安全性和效力，但不同生产平台的临床结果是一致的。了解这些平台的细微差别仍将为临床医生监督基于aav的治疗提供有价值的见解。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Medizinische Genetik GENETICS & HEREDITY-

自引率

9.10%

发文量

期刊介绍： medizinischegenetik is a scientific journal that is owned and published by the German Society of Human Genetics e.V. since 1989. The journal was founded by Prof. Jan Murken, München. Self-published until 2006, from 2007-2019 published at Springer Verlag and since 2020 at De Gruyter. medizinischegenetik serves education and training among colleagues, the interdisciplinary exchange of knowledge in all areas of human genetics in clinics, practice, research and teaching. Each issue of the quarterly journal deals with a focus that provides a comprehensive overview of current developments in specific clinical pictures, technical developments and therapeutic approaches. All reviews are written in English language. The journal thus creates a platform for the international exchange of knowledge and increased awareness of German research activities in the scientific community. In addition, medizinischegenetik contains information on activities in its own subject in the German-language section. This includes conference reports, association announcements, personnel matters, statements and guidelines. With health policy questions, historical retrospectives and comments on current developments, the profession takes a stand on human genetic issues in Germany, Austria and Switzerland.