Hoban Megan D, Bauer Daniel E
Division of Hematology/Oncology, Boston Children's Hospital, Boston, MA; Department of Pediatric Oncology, Dana-Farber Cancer Institute, Boston, MA; and Department of Pediatrics, Harvard Medical School, Harvard Stem Cell Institute, Boston, MA.
Blood. 2016 May 26;127(21):2525-35. doi: 10.1182/blood-2016-01-678151. Epub 2016 Apr 6.
Gene editing enables the site-specific modification of the genome. These technologies have rapidly advanced such that they have entered common use in experimental hematology to investigate genetic function. In addition, genome editing is becoming increasingly plausible as a treatment modality to rectify genetic blood disorders and improve cellular therapies. Genome modification typically ensues from site-specific double-strand breaks and may result in a myriad of outcomes. Even single-strand nicks and targeted biochemical modifications that do not permanently alter the DNA sequence (epigenome editing) may be powerful instruments. In this review, we examine the various technologies, describe their advantages and shortcomings for engendering useful genetic alterations, and consider future prospects for genome editing to impact hematology.
基因编辑能够对基因组进行位点特异性修饰。这些技术发展迅速，已在实验血液学中普遍用于研究基因功能。此外，作为纠正遗传性血液疾病和改进细胞疗法的一种治疗方式，基因组编辑正变得越来越可行。基因组修饰通常源于位点特异性双链断裂，可能会导致无数种结果。即使是不会永久改变DNA序列的单链切口和靶向生化修饰（表观基因组编辑）也可能是强大的工具。在本综述中，我们研究了各种技术，描述了它们在产生有用的基因改变方面的优缺点，并考虑了基因组编辑对血液学产生影响的未来前景。
