© Springer International Publishing AG 2017 3
S.H. Tsang (ed.), Precision Medicine, CRISPR, and Genome Engineering,
Advances in Experimental Medicine and Biology 1016,
DOI 10.1007/978-3-319-63904-8_
Chapter 1
Viral Vectors, Engineered Cells
and the CRISPR Revolution
James E. DiCarlo, Anurag Deeconda, and Stephen H. Tsang
Abstract Over the past few decades the ability to edit human cells has revolution-
ized modern biology and medicine. With advances in genome editing methodolo-
gies, 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 sys-
tems derived from prokaryotic immune systems has allowed for precision in cor-
recting various disease mutations. Furthermore, delivery of genetic payloads by
employing viral tropism has become a crucial and effective mechanism for deliver-
ing 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.
Keywords CRISPR/Cas • Ophthalmology • Genome Surgery • Gene Therapy
J.E. DiCarlo, Ph.D. (*) • A. Deeconda
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
e-mail: [email protected]; [email protected]
S.H. Tsang, M.D., Ph.D. (*)
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
e-mail: [email protected]