In the next decades, gene editing technologies are expected to be used in the treatment and prevention of human diseases. Yet, the future uses of gene editing in medicine are still unknown, including its applicability and effectiveness to the treatment and prevention of infectious diseases, cancer, and monogenic and polygenic hereditary diseases. This study aims to address this gap by analyzing the views of over 1,000 gene editing-related researchers from all over the world. Some of our survey results show that, in the next 10 years, DNA double-strand breaks are expected to be the main method for gene editing, and CRISPR-Cas systems to be the mainstream programmable nuclease. In the same period, gene editing is expected to have more applicability and effectiveness to treat and prevent infectious diseases and cancer. Off-targeting mutations, reaching therapeutic levels of editing efficiency, difficulties in targeting specific tissues in vivo, and regulatory and ethical challenges are among the most relevant factors that might hamper the use of gene editing in humans. In conclusion, our results suggest that gene editing might become a reality to the treatment and prevention of a variety of human diseases in the coming 10 years. If the future confirms these researchers' expectations, gene editing could change the way medicine, health systems, and public health deal with the treatment and prevention of human diseases.

中文翻译：

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用于治疗和预防人类疾病的基因编辑：对基因编辑相关研究人员的全球调查。

在接下来的几十年里，基因编辑技术有望用于人类疾病的治疗和预防。然而，基因编辑在医学中的未来用途仍然未知，包括其在治疗和预防传染病、癌症以及单基因和多基因遗传病方面的适用性和有效性。本研究旨在通过分析来自世界各地的 1,000 多名基因编辑相关研究人员的观点来弥补这一差距。我们的一些调查结果表明，在未来 10 年中，DNA 双链断裂有望成为基因编辑的主要方法，而 CRISPR-Cas 系统将成为主流的可编程核酸酶。同期，基因编辑有望在治疗和预防传染病和癌症方面具有更大的适用性和有效性。脱靶突变，体内、监管和伦理挑战是可能阻碍基因编辑在人类中使用的最相关因素之一。总之，我们的研究结果表明，基因编辑可能会在未来 10 年内成为治疗和预防各种人类疾病的现实。如果未来证实了这些研究人员的预期，基因编辑可能会改变医学、卫生系统和公共卫生处理和预防人类疾病的方式。