Horae Gene Therapy Center and Department of Pediatrics, University of Massachusetts Medical School, Worcester, Massachusetts 01655, USA; email:
Annu Rev Virol. 2019 Sep 29;6(1):601-621. doi: 10.1146/annurev-virology-092818-015530. Epub 2019 Jul 5.
The recent market approvals of recombinant adeno-associated virus (rAAV) gene therapies in Europe and the United States are landmark achievements in the history of modern science. These approvals are also anticipated to herald the emergence of a new class of therapies for monogenic disorders, which had hitherto been considered untreatable. These events can be viewed as stemming from the convergence of several important historical trends: the study of basic virology, the development of genomic technologies, the imperative for translational impact of National Institutes of Health-funded research, and the development of economic models for commercialization of rare disease therapies. In this review, these historical trends are described and the key developments that have enabled clinical rAAV gene therapies are discussed, along with an overview of the current state of the field and future directions.
近年来，重组腺相关病毒（rAAV）基因疗法在欧美获得批准，这是现代科学史上的里程碑式成就。这些批准预计也将预示着一类针对单基因疾病的新疗法的出现，而这些疾病此前被认为是无法治疗的。这些事件可以被视为源自几个重要历史趋势的融合：基础病毒学研究、基因组技术的发展、美国国立卫生研究院资助的研究转化为实际影响的必要性，以及罕见病疗法商业化的经济模式的发展。在这篇综述中，描述了这些历史趋势，并讨论了促成临床 rAAV 基因疗法的关键进展，以及该领域的现状和未来方向。
