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.

中文翻译：

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Luxturna（以及 Zolgensma 和 Glybera）的重组腺相关病毒基因治疗：我们在哪里，我们是如何到达这里的？


最近，重组腺相关病毒（rAAV）基因疗法在欧洲和美国获得市场批准，是现代科学史上的里程碑式成就。这些批准预计也预示着一类新的单基因疾病疗法的出现，迄今为止单基因疾病一直被认为是无法治疗的。这些事件可以被视为源于几个重要历史趋势的融合：基础病毒学的研究、基因组技术的发展、美国国立卫生研究院资助的研究转化影响的必要性以及商业化经济模型的发展的罕见疾病疗法。在这篇综述中，描述了这些历史趋势，讨论了实现临床 rAAV 基因治疗的关键进展，并概述了该领域的现状和未来方向。