评估和推进 CRISPR-Cas 工具的安全性：从 DNA 到 RNA 编辑。

Department of Pathology, Boston Children's Hospital and Harvard Medical School, Boston, MA, 02115, USA.

Division of Hematology/Oncology, Boston Children's Hospital, Department of Pediatric Oncology, Dana-Farber Cancer Institute, Harvard Stem Cell Institute, Broad Institute, Department of Pediatrics, Harvard Medical School, Boston, MA, 02115, USA.

Nat Commun. 2023 Jan 13;14(1):212. doi: 10.1038/s41467-023-35886-6.

CRISPR-Cas gene editing has revolutionized experimental molecular biology over the past decade and holds great promise for the treatment of human genetic diseases. Here we review the development of CRISPR-Cas9/Cas12/Cas13 nucleases, DNA base editors, prime editors, and RNA base editors, focusing on the assessment and improvement of their editing precision and safety, pushing the limit of editing specificity and efficiency. We summarize the capabilities and limitations of each CRISPR tool from DNA editing to RNA editing, and highlight the opportunities for future improvements and applications in basic research, as well as the therapeutic and clinical considerations for their use in patients.

CRISPR-Cas 基因编辑在过去十年中彻底改变了实验分子生物学，为治疗人类遗传疾病带来了巨大的希望。在这里，我们回顾了 CRISPR-Cas9/Cas12/Cas13 核酸酶、DNA 碱基编辑器、prime 编辑器和 RNA 碱基编辑器的发展，重点评估和改进了它们的编辑精度和安全性，推动了编辑特异性和效率的极限。我们总结了每种 CRISPR 工具从 DNA 编辑到 RNA 编辑的能力和局限性，并强调了在基础研究中进一步改进和应用的机会，以及在患者中使用时的治疗和临床考虑因素。