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基因组编辑与血液系统恶性肿瘤。

Genome Editing and Hematologic Malignancy.

机构信息

Division of Hospital Medicine, Department of Internal Medicine and Life Sciences Institute, University of Michigan, Ann Arbor, Michigan 48109, USA; email:

Departments of Internal Medicine, Human Genetics, and Pediatrics, Life Sciences Institute, and Howard Hughes Medical Institute, University of Michigan, Ann Arbor, Michigan 48109, USA; email:

出版信息

Annu Rev Med. 2020 Jan 27;71:71-83. doi: 10.1146/annurev-med-052318-100741. Epub 2019 Aug 30.

Abstract

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.

摘要

现代基因组时代见证了我们对疾病分子基础理解的显著进步,但将基础发现转化为新的疾病治疗方法的进展却相对滞后。最近,基因组编辑技术的突破为其直接治疗疾病遗传原因的潜力带来了希望。与任何治疗干预一样,基因组编辑应该根据其潜在的风险和益处来考虑。在这篇综述中,我们强调了基因组编辑疗法的前景,以及其临床应用面临的概念和技术障碍,特别强调了血液系统恶性肿瘤。

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