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用于基因治疗的病毒载体系统:进展与生物安全挑战的综合文献综述

Viral Vector Systems for Gene Therapy: A Comprehensive Literature Review of Progress and Biosafety Challenges.

作者信息

Ghosh Sumit, Brown Alex M, Jenkins Chris, Campbell Katie

机构信息

The Abigail Wexner Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.

Clinical Biosafety Services, A Division of Sabai Global, Wildwood, MO, USA.

出版信息

Appl Biosaf. 2020 Mar 1;25(1):7-18. doi: 10.1177/1535676019899502.

Abstract

INTRODUCTION

National Institutes of Health (NIH) defines gene therapy as an experimental technique that uses genes to treat or prevent disease. Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be effective and safe.

METHODS

Applications of viral vectors and nonviral gene delivery systems have found an encouraging new beginning in gene therapy in recent years. Although several viral vectors and nonviral gene delivery systems have been developed in the past 3 decades, no one delivery system can be applied in gene therapy to all cell types in vitro and in vivo. Furthermore, the use of viral vector systems (both in vitro and in vivo) present unique occupational health and safety challenges. In this review article, we discuss the biosafety challenges and the current framework of risk assessment for working with the viral vector systems.

DISCUSSION

The recent advances in the field of gene therapy is exciting, but it is important for scientists, institutional biosafety committees, and biosafety officers to safeguard public trust in the use of this technology in clinical trials and make conscious efforts to engage the public through ongoing forums and discussions.

摘要

引言

美国国立卫生研究院(NIH)将基因治疗定义为一种利用基因治疗或预防疾病的实验技术。尽管基因治疗对于多种疾病(包括遗传性疾病、某些类型的癌症和特定病毒感染)是一种有前景的治疗选择,但该技术仍然存在风险,仍在研究以确保其有效性和安全性。

方法

近年来,病毒载体和非病毒基因递送系统的应用在基因治疗领域有了令人鼓舞的新开端。尽管在过去30年中已经开发了几种病毒载体和非病毒基因递送系统,但没有一种递送系统可以在体外和体内的基因治疗中应用于所有细胞类型。此外,病毒载体系统的使用(无论是在体外还是体内)都带来了独特的职业健康和安全挑战。在这篇综述文章中,我们讨论了使用病毒载体系统的生物安全挑战和当前的风险评估框架。

讨论

基因治疗领域的最新进展令人兴奋,但对于科学家、机构生物安全委员会和生物安全官员来说,在临床试验中保障公众对这项技术使用的信任,并通过持续的论坛和讨论有意识地与公众互动非常重要。

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