Gene therapies are currently one of the most investigated therapeutic modalities in both the preclinical and clinical settings and have shown promise in treating a diverse spectrum of diseases. Gene therapies aim at introducing a gene material in target cells and represent a promising approach to cure diseases that were thought to be incurable by conventional modalities. In many cases, a gene therapy requires a vector to deliver gene therapeutics into target cells; viral vectors are among the most widely studied vectors owing to their distinguished advantages such as outstanding transduction efficiency. With decades of development, viral vector-based gene therapies have achieved promising clinical outcomes with many products approved for treating a range of diseases including cancer, infectious diseases and monogenic diseases. In addition, a number of active clinical trials are underway to further expand their therapeutic potential. In this review, we highlight the diversity of viral vectors, review approved products, and discuss the current clinical landscape of in vivo viral vector-based gene therapies. We have reviewed 13 approved products and their clinical applications. We have also analyzed more than 200 active trials based on various viral vectors and discussed their respective therapeutic applications. Moreover, we provide a critical analysis of the major translational challenges for in vivo viral vector-based gene therapies and discuss possible strategies to address the same.

中文翻译：

---

临床中基于病毒载体的基因疗法


基因疗法是目前在临床前和临床环境中研究最多的治疗方式之一，并在治疗多种疾病方面显示出前景。基因疗法旨在将基因材料引入靶细胞，并代表了一种有前途的方法来治疗传统疗法无法治愈的疾病。在许多情况下，基因疗法需要载体将基因疗法传递到靶细胞中；病毒载体因其出色的转导效率等显着优点而成为研究最广泛的载体之一。经过几十年的发展，基于病毒载体的基因疗法已经取得了可喜的临床成果，许多产品被批准用于治疗一系列疾病，包括癌症、传染病和单基因疾病。此外，许多积极的临床试验正在进行中，以进一步扩大其治疗潜力。在这篇综述中，我们强调了病毒载体的多样性，回顾了批准的产品，并讨论了基于体内病毒载体的基因疗法的当前临床前景。我们审查了 13 个批准的产品及其临床应用。我们还分析了 200 多项基于各种病毒载体的活跃试验，并讨论了它们各自的治疗应用。此外，我们对基于体内病毒载体的基因疗法的主要转化挑战进行了批判性分析，并讨论了解决这些挑战的可能策略。