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

vii

Part I Introduction to the CRISPR Revolution

1 Viral Vectors, Engineered Cells and the CRISPR Revolution....... 3

James E. DiCarlo, Anurag Deeconda, and Stephen H. Tsang

2 Combining Engineered Nucleases with Adeno- associated

Viral Vectors for Therapeutic Gene Editing...................... 29

Benjamin E. Epstein and David V. Schaffer

Part II CRISPR in Model Systems

3 From Reductionism to Holism: Toward a More Complete

View of Development Through Genome Engineering.............. 45

Rebecca K. Delker and Richard S. Mann

4 A Transgenic Core Facility’s Experience in Genome

Editing Revolution.......................................... 75

Celvie L. Yuan and Yueh-Chiang Hu

5 Genome Editing to Study Ca2+ Homeostasis

in Zebrafish Cone Photoreceptors.............................. 91

Susan E. Brockerhoff

6 CRISPR: From Prokaryotic Immune Systems

to Plant Genome Editing Tools................................ 101

Anindya Bandyopadhyay, Shamik Mazumdar, Xiaojia Yin,

and William Paul Quick

Contents