Department of Biomedicine, Aarhus University, Aarhus C, Denmark.
Aarhus Institute of Advanced Studies (AIAS), Aarhus University, Aarhus C, Denmark.
Br J Haematol. 2019 Jun;185(5):821-835. doi: 10.1111/bjh.15851. Epub 2019 Mar 12.
The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/CRISPR-associated (Cas)9 platform offers an efficient way of making precise genetic changes to the human genome. This can be employed for disruption, addition and correction of genes, thereby enabling a new class of genetic therapies that can be applied to haematological disorders. Here we review recent technological advances in the CRISPR/Cas9 methodology and applications in haematology for curing monogenic genetic disorders and for engineering novel chimeric antigen receptor (CAR) T cells to treat haematological malignancies. Furthermore, we discuss current challenges for full clinical implementation of CRISPR/Cas9, and reflect on future trajectories of the technology.
簇状规律间隔短回文重复序列 (CRISPR)/CRISPR 相关 (Cas)9 平台为人类基因组进行精确的遗传改变提供了一种有效的方法。这可以用于破坏、添加和纠正基因，从而实现一类新的遗传疗法，可应用于血液疾病。在这里，我们回顾了 CRISPR/Cas9 方法在血液学中的最新技术进展及其在治疗单基因遗传疾病和工程新型嵌合抗原受体 (CAR) T 细胞以治疗血液恶性肿瘤方面的应用。此外，我们还讨论了 CRISPR/Cas9 完全临床应用的当前挑战，并思考了该技术的未来发展轨迹。
