UCL School of Pharmacy, University College London, London WC1N 1AX, UK.
Wits/SAMRC Antiviral Gene Therapy Research Unit, Faculty of Health Sciences, University of the Witwatersrand, Johannesburg 2193, South Africa.
Biomolecules. 2021 Apr 20;11(4):611. doi: 10.3390/biom11040611.
Rare monogenic disorders such as lysosomal diseases have been at the forefront in the development of novel treatments where therapeutic options are either limited or unavailable. The increasing number of successful pre-clinical and clinical studies in the last decade demonstrates that gene therapy represents a feasible option to address the unmet medical need of these patients. This article provides a comprehensive overview of the current state of the field, reviewing the most used viral gene delivery vectors in the context of lysosomal storage disorders, a selection of relevant pre-clinical studies and ongoing clinical trials within recent years.
罕见的单基因疾病，如溶酶体贮积症，一直处于新型治疗方法的前沿，这些疾病的治疗选择有限或不可用。在过去十年中，越来越多的成功的临床前和临床研究表明，基因治疗是解决这些患者未满足的医疗需求的可行选择。本文全面概述了该领域的现状，回顾了溶酶体贮积症背景下最常用的病毒基因传递载体，选择了近年来相关的临床前研究和正在进行的临床试验。
