Genome Editing. A Practical Guide to Research and Clinical Applications

Table of Contents

1. Basic principles of genome editing2. Choosing a target site and editing strategy3. Choosing a nuclease, guide RNA, and repair template4. Assessing for off-target mutagenesis5. Assessing the outcomes of genome editing6. Base editing and other advanced forms of genome editing7. Epigenome editing8. Cellular disease modeling and risk prediction9. Functional experiments and screens10. Therapeutic applications11. Ethical implications

Authors

Kiran Musunuru Associate Professor of Cardiovascular Medicine and Genetics, Perelman School of Medicine, University of Pennsylvania, USA. Kiran Musunuru, MD, PhD, MPH, ML received his medical degree from Weill Cornell Medical College, his PhD from The Rockefeller University, and his Master of Public Health from Johns Hopkins Bloomberg School of Public Health. He trained in Internal Medicine at Brigham and Women's Hospital and Cardiovascular Medicine at Johns Hopkins Hospital, followed by postdoctoral work at Massachusetts General Hospital and the Broad Institute of MIT and Harvard. Dr Musunuru's research focuses on the genetics of cardiovascular and metabolic diseases and seeks to identify naturally occurring genetic variants that predispose to or protect against disease and can be used to develop therapies to protect the entire population. His expertise includes the use of human pluripotent stem cells as a platform for disease modelling and the use of genome-editing tools such as CRISPR-Cas9 for research and therapeutic applications. In 2016, he received the Presidential Early Career Award for Scientists and Engineers from U.S. President Obama, as well as the American Heart Association's Award of Meritorious Achievement. He became Editor-in-Chief of Circulation: Genomic and Precision Medicine (an American Heart Association journal) in 2018.