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The present and future of genome editing in cancer research

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Abstract

The widespread use of high-throughput genome sequencing methods is profoundly changing the way we understand, classify, and treat human cancers. To make sense of the deluge of sequencing data generated in the clinic, more effective and rapid assessments of the functional relevance of newly discovered cancer-associated mutations are urgently needed. In this review, we discuss how genome editing technologies are responding to this major challenge. Largely focusing on CRISPR-based methods, we will highlight their potential to accelerate discovery, discuss their current limitations, and speculate about future applications.

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Acknowledgments

We thank members of the Ventura lab for comments and suggestions, Jennifer Hollenstein for editing the manuscript, and Yanlan Huang for assistance in generating the figure. We apologize to our colleagues whose work we could not cite due to space limitation. Work on genome editing in the Ventura lab is supported by the MSK Cancer Center Support Grant/Core Grant (P30 CA008748) and by grants from the Geoffrey Beene Cancer Research Foundation, the Uniting Against Lung Cancer Foundation, the Cycle for Survival Foundation, and the Pershing Square Sohn Cancer Research Alliance.

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  1. Cancer Biology and Genetics Program, Memorial Sloan Kettering Cancer Center, New York, NY, USA

    Xiaoyi Li, Raymond Wu & Andrea Ventura

  2. Gerstner Sloan Kettering Graduate School of Biomedical Sciences, New York, NY, USA

    Xiaoyi Li & Raymond Wu

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  1. Xiaoyi Li
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  2. Raymond Wu
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Correspondence to Andrea Ventura.

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Li, X., Wu, R. & Ventura, A. The present and future of genome editing in cancer research. Hum Genet 135, 1083–1092 (2016). https://doi.org/10.1007/s00439-016-1713-3

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