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Annu Rev Med. 2019 Aug 30. doi: 10.1146/annurev-med-052318-100741. [Epub ahead of print]

Genome Editing and Hematologic Malignancy.

Author information

1
Division of Hospital Medicine, Department of Internal Medicine and Life Sciences Institute, University of Michigan, Ann Arbor, Michigan 48109, USA; email: bemmer@med.umich.edu.
2
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: ginsburg@umich.edu.

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. Expected final online publication date for the Annual Review of Medicine, Volume 71 is January 27, 2020. Please see http://www.annualreviews.org/page/journal/pubdates for revised estimates.

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