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Viral Vectors, Engineered Cells and the CRISPR Revolution

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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 decades the ability to edit human cells has revolutionized modern biology and medicine. With advances in genome editing methodologies, gene delivery and cell-based therapeutics targeted at treatment of genetic disease have become a reality that will become more and more essential in clinical practice. Modifying specific mutations in eukaryotic cells using CRISPR-Cas systems derived from prokaryotic immune systems has allowed for precision in correcting various disease mutations. Furthermore, delivery of genetic payloads by employing viral tropism has become a crucial and effective mechanism for delivering genes and gene editing systems into cells. Lastly, cells modified ex vivo have tremendous potential and have shown effective in studying and treating a myriad of diseases. This chapter seeks to highlight and review important progress in the realm of the editing of human cells using CRISPR-Cas systems, the use of viruses as vectors for gene therapy, and the application of engineered cells to study and treat disease.

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Authors and Affiliations

  1. Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA

    James E. DiCarlo Ph.D. & Anurag Deeconda

  2. Department of Pathology and Cell Biology, Institute of Human Nutrition, College of Physicians, Columbia University, New York, NY, USA

    James E. DiCarlo Ph.D. & Anurag Deeconda

  3. Jonas Children’s Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY, USA

    James E. DiCarlo Ph.D. & Anurag Deeconda

  4. Institute of Human Nutrition, College of Physicians and Surgeons, Columbia University, New York, NY, USA

    Stephen H. Tsang M.D., Ph.D.

  5. Department of Ophthalmology, Columbia University, New York, NY, USA

    Stephen H. Tsang M.D., Ph.D.

  6. Jonas Children’s Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Columbia University, New York, NY, USA

    Stephen H. Tsang M.D., Ph.D.

  7. Department of Pathology and Cell Biology, Columbia University, New York, NY, USA

    Stephen H. Tsang M.D., Ph.D.

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  1. James E. DiCarlo Ph.D.
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Correspondence to James E. DiCarlo 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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DiCarlo, J.E., Deeconda, A., Tsang, S.H. (2017). Viral Vectors, Engineered Cells and the CRISPR Revolution. 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_1

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