Viral Vectors, Engineered Cells and the CRISPR Revolution.

James E DiCarlo, Anurag Deeconda, Stephen H Tsang

Journal: Advances in experimental medicine and biology 2018;1016():3-27

PMID: 29130151

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."]

Address: Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA. [email protected].; Department of Pathology and Cell Biology, Institute of Human Nutrition, College of Physicians, Columbia University, New York, NY, USA. [email protected].; Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY, USA. [email protected].; Edward S. Harkness Eye Institute, New York-Presbyterian Hospital, New York, NY, USA.; Department of Pathology and Cell Biology, Institute of Human Nutrition, College of Physicians, Columbia University, New York, NY, USA.; Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Department of Ophthalmology, Columbia University Medical Center, New York, NY, USA.; Institute of Human Nutrition, College of Physicians and Surgeons, Columbia University, New York, NY, USA.; Department of Ophthalmology, Columbia University, New York, NY, USA.; Jonas Children's Vision Care, and Bernard & Shirlee Brown Glaucoma Laboratory, Columbia University, New York, NY, USA.; Department of Pathology and Cell Biology, Columbia University, New York, NY, USA.

DOI: 10.1007/978-3-319-63904-8_1

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https://dx.doi.org/10.1007/978-3-319-63904-8_1

MeSH Terms: CRISPR-Cas Systems, Cell Engineering, Gene Editing, Genetic Therapy, Genetic Vectors, Humans, Viruses