Synthetic Biology: Emerging Concepts to Design and Advance Adeno‐Associated Viral Vectors for Gene Therapy,Advanced Science

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Synthetic Biology: Emerging Concepts to Design and Advance Adeno‐Associated Viral Vectors for Gene Therapy
Advanced Science ( IF 14.3 ) Pub Date : 2021-02-26 , DOI: 10.1002/advs.202004018
Hanna J Wagner _{1,

2,

3} , Wilfried Weber _{2,

3} , Martin Fussenegger _{1,

4}

Affiliation

Three recent approvals and over 100 ongoing clinical trials make adeno‐associated virus (AAV)‐based vectors the leading gene delivery vehicles in gene therapy. Pharmaceutical companies are investing in this small and nonpathogenic gene shuttle to increase the therapeutic portfolios within the coming years. This prospect of marking a new era in gene therapy has fostered both investigations of the fundamental AAV biology as well as engineering studies to enhance delivery vehicles. Driven by the high clinical potential, a new generation of synthetic‐biologically engineered AAV vectors is on the rise. Concepts from synthetic biology enable the control and fine‐tuning of vector function at different stages of cellular transduction and gene expression. It is anticipated that the emerging field of synthetic‐biologically engineered AAV vectors can shape future gene therapeutic approaches and thus the design of tomorrow's gene delivery vectors. This review describes and discusses the recent trends in capsid and vector genome engineering, with particular emphasis on synthetic‐biological approaches.

中文翻译：

合成生物学：设计和推进用于基因治疗的腺相关病毒载体的新兴概念

最近的三项批准和超过 100 项正在进行的临床试验使基于腺相关病毒 (AAV) 的载体成为基因治疗中领先的基因递送载体。制药公司正在投资这种小型非致病性基因穿梭，以在未来几年内增加治疗组合。这种标志着基因治疗新时代的前景促进了对 AAV 基础生物学的研究以及增强递送载体的工程研究。在巨大临床潜力的推动下，新一代合成生物工程 AAV 载体正在兴起。合成生物学的概念使得能够在细胞转导和基因表达的不同阶段控制和微调载体功能。预计合成生物工程 AAV 载体这一新兴领域可以塑造未来的基因治疗方法，从而塑造未来基因递送载体的设计。这篇综述描述并讨论了衣壳和载体基因组工程的最新趋势，特别强调合成生物学方法。

更新日期：2021-05-05

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