Precision Medicine, CRISPR, and Genome Engineering Moving from Association to Biology and Therapeutics

Advances in Experimental Medicine and Biology 1016 Stephen H. Tsang Editor Precision Medicine, CRISPR, and Genome Engineering Mo ...

Advances in Experimental Medicine and Biology Editorial Board: IRUN R. COHEN, The Weizmann Institute of Science, Rehovot, Israel ...

Stephen H. Tsang Editor Precision Medicine, CRISPR, and Genome Engineering Moving from Association to Biology and Therapeutics F ...

ISSN 0065-2598 ISSN 2214-8019 (electronic) Advances in Experimental Medicine and Biology ISBN 978-3-319-63903-1 ISBN 978-3-319-6 ...

v Foreword All of life is encoded in the four letters of DNA, ATGC. Recent advances in genome engineering now enable us to manip ...

vii Part I Introduction to the CRISPR Revolution 1 Viral Vectors, Engineered Cells and the CRISPR Revolution....... 3 James E. D ...

viii Part III The Future of CRISPR 7 Target Discovery for Precision Medicine Using High-Throughput Genome Engineering........... ...

ix About the Authors Stephen H.  Tsang For years, Stephen H. Tsang has been culturing embryonic stem (ES) cells and created the ...

Part I Introduction to the CRISPR Revolution ...

© Springer International Publishing AG 2017 3 S.H. Tsang (ed.), Precision Medicine, CRISPR, and Genome Engineering, Advances in ...

4 1.1 Introduction Precise engineering of human cells using genetic tools has revolutionized biology. Frederick Griffith’s obser ...

5 Cellular manipulation has produced engineered cells with great therapeutic potential. Notably, the use of chimeric antigen rec ...

6 These endonucleases are highly efficient and have been used in the modification of human cells as well as numerous other eukar ...

7 1.2.2 CRISPR-Cas Systems While both ZFNs and TALENs are highly used in biomedicine, their individual disadvantages have led re ...

8 Cas9 to remove all porcine endogenous retroviruses from a porcine epithelial cell line, with the ultimate future goal of safe ...

9 structural polyprotein, and the env gene encodes envelope proteins essential in bind- ing to host cells and determining viral ...

10 the nuclease can recombine during the reverse transcription of the viral genome [ 72 ]. However, using a similar approach as ...

11 viral and host factors. These genes are often removed or manipulated to both make room for transgenes as well as decrease the ...

12 rupt the CCR5 locus of CD4+ T cells with high frequency, making them resistant to HIV-1 infection [ 102 ]. However unlike len ...

13 cocaine and nicotine [ 116 ]. By covalently linking small molecule analogs of such addictive compounds, De et  al. have used ...