基因治疗代表着在纠正遗传突变、产生细胞系和动物模型以及改进癌症免疫治疗的可行性和效果方面，对血液学领域进行根本性革命的重大潜力。与其他不同的基因工程工具相比，CRISPR相关蛋白9（Cas9）成为一种有效和多功能的基因编辑器，能够精确修改基因组。在各种血液学疾病中，基因工程的应用显示出令人鼓舞的结果。单基因遗传性血液学疾病可以通过单个基因修改来纠正。通过使用CRISPR-CAS9，在镰刀细胞贫血、β-地中海贫血和血友病疾患中成功实现了功能性红细胞和止血因子的恢复。我们通过CRISPR-CAS9技术提高了对血液肿瘤学的理解。CRISPR-CAS9有助于建立一个突变基因平台，该平台能够调控白血病中细胞存活和增殖。CRISPR-CAS9在多发性骨髓瘤和急性淋巴细胞白血病中与嵌合抗原受体（CAR）T细胞疗法相结合时，基因编辑技术的治疗应用是可行的，并且能够减轻CAR-T细胞疗法的缺陷。本文概述了CRISPR-Cas9在β-血红蛋白病和血友病治疗中的最新文献。我们介绍了所采用的策略以及临床前和临床试验的研究结果。此外，本文将讨论基因工程在血液肿瘤学领域作为促进和克服嵌合抗原受体T细胞疗法（CAR-T）的缺点的适当工具。© 2023. 作者个人, 由 Springer Verlag GmbH Germany 以独家许可的形式提供，属于 Springer Nature 公司的一部分。

Gene therapy represents a significant potential to revolutionize the field of hematology with applications in correcting genetic mutations, generating cell lines and animal models, and improving the feasibility and efficacy of cancer immunotherapy. Compared to different genetic engineering tools, clustered regularly interspaced short palindromic repeats (CRISPR) CRISPR-associated protein 9 (Cas9) emerged as an effective and versatile genetic editor with the ability to precisely modify the genome. The applications of genetic engineering in various hematological disorders have shown encouraging results. Monogenic hematological disorders can conceivably be corrected with single gene modification. Through the use of CRISPR-CAS9, restoration of functional red blood cells and hemostasis factors were successfully attained in sickle cell anemia, beta-thalassemia, and hemophilia disorders. Our understanding of hemato-oncology has been advanced via CRIPSR-CAS9 technology. CRISPR-CAS9 aided to build a platform of mutated genes responsible for cell survival and proliferation in leukemia. Therapeutic application of CRISPR-CAS9 when combined with chimeric antigen receptor (CAR) T cell therapy in multiple myeloma and acute lymphoblastic leukemia was feasible with attenuation of CAR T cell therapy pitfalls. Our review outlines the latest literature on the utilization of CRISPR-Cas9 in the treatment of beta-hemoglobinopathies and hemophilia disorders. We present the strategies that were employed and the findings of preclinical and clinical trials. Also, the review will discuss gene engineering in the field of hemato-oncology as a proper tool to facilitate and overcome the drawbacks of chimeric antigen receptor T cell therapy (CAR-T).© 2023. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.