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Target Discovery for Precision Medicine Using High-Throughput Genome Engineering

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Precision Medicine, CRISPR, and Genome Engineering

Part of the book series: Advances in Experimental Medicine and Biology ((AEMB,volume 1016))

Abstract

Over the past few years, programmable RNA-guided nucleases such as the CRISPR/Cas9 system have ushered in a new era of precision genome editing in diverse model systems and in human cells. Functional screens using large libraries of RNA guides can interrogate a large hypothesis space to pinpoint particular genes and genetic elements involved in fundamental biological processes and disease-relevant phenotypes. Here, we review recent high-throughput CRISPR screens (e.g. loss-of-function, gain-of-function, and targeting noncoding elements) and highlight their potential for uncovering novel therapeutic targets, such as those involved in cancer resistance to small molecular drugs and immunotherapies, tumor evolution, infectious disease, inborn genetic disorders, and other therapeutic challenges.

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Authors and Affiliations

  1. New York Genome Center, 101 Avenue of the Americas, New York, NY, 10013, USA

    Xinyi Guo, Poonam Chitale & Neville E. Sanjana Ph.D.

  2. Department of Biology, New York University, New York, NY, 10003, USA

    Xinyi Guo, Poonam Chitale & Neville E. Sanjana Ph.D.

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  1. Xinyi Guo
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  2. Poonam Chitale
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  3. Neville E. Sanjana Ph.D.
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Correspondence to Neville E. Sanjana Ph.D. .

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  1. Departments of Ophthalmology, Pathology & Cell Biology and Biomedical Engineering, Institute of Human Nutrition, Columbia Stem Cell Initiative, Herbert Irving Comprehensive Cancer Center, Graduate Program in Neurobiology & Behavior, Columbia University, New York, New York, USA

    Stephen H. Tsang

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Guo, X., Chitale, P., Sanjana, N.E. (2017). Target Discovery for Precision Medicine Using High-Throughput Genome Engineering. In: Tsang, S. (eds) Precision Medicine, CRISPR, and Genome Engineering. Advances in Experimental Medicine and Biology, vol 1016. Springer, Cham. https://doi.org/10.1007/978-3-319-63904-8_7

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