美国加州大学伯克利分校Jennifer A. Doudna综述了治疗性基因组编辑的前景与挑战。这一成果发表在2020年2月12日的国际学术期刊《自然》上。

文中讨论，将CRISPR（聚类的规则间隔的回文短重复序列）技术用于人类治疗的科学、技术和道德方面，重点是突出机遇和挑战的具体实例。基因组编辑已经或即将在临床上用于多种疾病，并且正在开发更多的应用程序。该领域的快速发展要求积极努力确保将该突破性技术可靠地用于治疗、治愈和预防遗传疾病。

据了解，基因组编辑涉及对细胞DNA序列的精确操纵，以改变细胞命运和生物特征，具有提高人们对人类遗传学认识和治愈遗传疾病的潜力。

附：英文原文

Title: The promise and challenge of therapeutic genome editing

Author: Jennifer A. Doudna

Issue&Volume: 2020-02-12

Abstract: Genome editing, which involves the precise manipulation of cellular DNA sequences to alter cell fates and organism traits, has the potential to both improve our understanding of human genetics and cure genetic disease. Here I discuss the scientific, technical and ethical aspects of using CRISPR (clustered regularly interspaced short palindromic repeats) technology for therapeutic applications in humans, focusing on specific examples that highlight both opportunities and challenges. Genome editing is—or will soon be—in the clinic for several diseases, with more applications under development. The rapid pace of the field demands active efforts to ensure that this breakthrough technology is used responsibly to treat, cure and prevent genetic disease.

DOI: 10.1038/s41586-020-1978-5

Source: https://www.nature.com/articles/s41586-020-1978-5