Genome Editing

A Practical Guide to Research and Clinical Applications

Häftad, Engelska, 2021

AvKiran Musunuru,USA) Musunuru, Kiran (Associate Professor of Cardiovascular Medicine and Genetics, Perelman School of Medicine, University of Pennsylvania

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Genome Editing: A Practical Guide to Research and Clinical Applications is geared towards investigators interested in learning how to use CRISPR-Cas9-based technologies, with a focus on cardiovascular research and clinical applications. Covering a range of topics from the basics of genome editing to design considerations, to assessments and applications, this reference allows readers to get started and establish a full workflow from the beginning of the project to its full completion. With worked examples drawn from real-life experiments, as well as troubleshooting and pitfalls to avoid, the book serves as an essential reference for researchers and investigators in both cardiovascular and biomedical research.

Help readers familiarise with the variety of genome-editing approaches that are being applied in cardiovascular research and medicine, i.e., both research applications and clinical applications
Understand the use of genome editing through worked examples (based on real-life experiments) in which CRISPR-Cas9 is employed, online tools to design CRISPR-Cas9 reagents, methods to interpret data from genome-editing experiments, the downsides of genome-editing technology - both the scientific and ethical pitfalls to avoid
Written in an easy-to-follow manner, guiding readers from the design of the project to its completion
Includes unpublished and new methods

Produktinformation

Utgivningsdatum2021-03-10
Mått191 x 235 x 18 mm
Vikt410 g
FormatHäftad
SpråkEngelska
Antal sidor230
FörlagElsevier Science
ISBN9780128234846

Tillhör följande kategorier

Biologi inom Naturvetenskap och teknik

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.

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