Gene therapies are gaining momentum as promising early successes in clinical studies accumulate and examples of regulatory approval for licensing increase. Investigators are advancing with cautious optimism that effective, durable, and safe therapies will provide benefit to patients-not only those with single-gene disorders but those with complex acquired diseases as well. While the strategies being translated from the lab to the clinic are numerous, this review focuses on the clinical research that has forged the gene therapy field as it currently stands.

中文翻译：

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进入基因治疗的现代时代。

随着有希望的临床研究早期成果的积累以及监管机构批准许可增加的例子，基因疗法正获得发展势头。研究人员正以谨慎的乐观态度前进，即有效，持久和安全的疗法将使患者受益，不仅是患有单基因疾病的患者，还包括患有复杂性获得性疾病的患者。尽管从实验室到临床的策略很多，但本文的重点是基因治疗领域的临床研究。