Engineering novel adeno-associated viruses (AAVs) for improved delivery in the nervous system

IF 6.9 2区 生物学 Q1 BIOCHEMISTRY & MOLECULAR BIOLOGY
Ana D. Carneiro , David V. Schaffer
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引用次数: 0

Abstract

Harnessing adeno-associated virus (AAV) vectors for therapeutic gene delivery has emerged as a progressively promising strategy to treat disorders of both the central nervous system (CNS) and peripheral nervous system (PNS), and there are many ongoing clinical trials. However, unique physiological and molecular characteristics of the CNS and PNS pose obstacles to efficient vector delivery, ranging from the blood-brain barrier to the diverse nature of nervous system disorders. Engineering novel AAV capsids may help overcome these ongoing challenges and maximize therapeutic transgene delivery. This article discusses strategies for innovative AAV capsid development, highlighting recent advances. Notably, advances in next generation sequencing and machine learning have sparked new approaches for capsid investigation and engineering. Furthermore, we outline future directions and additional challenges in AAV-mediated gene therapy in the CNS and PNS.
设计新型腺相关病毒 (AAV),改进在神经系统中的传输。
利用腺相关病毒(AAV)载体进行治疗性基因递送已逐渐成为治疗中枢神经系统(CNS)和周围神经系统(PNS)疾病的一种有前途的策略,目前有许多临床试验正在进行中。然而,从血脑屏障到神经系统疾病的多样性,中枢神经系统和外周神经系统独特的生理和分子特征给载体的高效传递造成了障碍。设计新型 AAV 外壳可能有助于克服这些持续存在的挑战,并最大限度地提高治疗性转基因递送。本文讨论了创新 AAV 病毒衣壳的开发策略,并重点介绍了最近取得的进展。值得注意的是,下一代测序和机器学习的进步为噬菌体研究和工程设计带来了新方法。此外,我们还概述了 AAV 介导的中枢神经系统和前枢神经系统基因治疗的未来方向和其他挑战。
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
Current Opinion in Chemical Biology
Current Opinion in Chemical Biology 生物-生化与分子生物学
CiteScore
13.30
自引率
1.30%
发文量
113
审稿时长
74 days
期刊介绍: COCHBI (Current Opinion in Chemical Biology) is a systematic review journal designed to offer specialists a unique and educational platform. Its goal is to help professionals stay informed about the growing volume of information in the field of Chemical Biology through systematic reviews.
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