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Genome Editing and Hematologic Malignancy.
Annual Review of Medicine ( IF 10.5 ) Pub Date : 2020-01-27 , DOI: 10.1146/annurev-med-052318-100741
Brian T Emmer 1 , David Ginsburg 2
Affiliation  

The modern genomic era has seen remarkable advancement in our understanding of the molecular basis for disease, yet translation of basic discoveries into new disease treatments has arguably lagged behind. Recently, breakthroughs in genome editing technologies have created hope for their potential to directly treat the genetic causes of disease. Like any therapeutic intervention, genome editing should be considered in light of its potential risks and benefits. In this review, we highlight the promise of genome editing therapies, as well as the conceptual and technical barriers to their clinical application, with a special emphasis on hematologic malignancies.

中文翻译:

基因组编辑和血液系统恶性肿瘤。

在我们对疾病的分子基础的理解上,现代基因组时代取得了显着进步,然而,将基本发现转化为新疾病的治疗却被认为落后了。最近,基因组编辑技术的突破为其直接治疗疾病的遗传原因的潜力创造了希望。像任何治疗性干预措施一样,应根据基因组编辑的潜在风险和益处来考虑基因组编辑。在这篇综述中,我们重点介绍了基因组编辑疗法的前景以及其临床应用的概念和技术障碍,并特别强调了血液系统恶性肿瘤。
更新日期:2020-04-21
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