Genome Editing

1. Front Matter

   Pages i-ix

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2. Genome Editing with Targetable Nucleases

   • Stephane Pelletier

   Pages 1-29

3. CRISPR/Cas9 Approaches to Investigate the Noncoding Genome

   • Davide Seruggia, Lluis Montoliu

   Pages 31-43

4. At the Conflux of Human Genome Engineering and Induced Pluripotency

   • Knut Woltjen, Fabian Oceguera-Yanez, Harunobu Kagawa, Shin-Il Kim

   Pages 45-64

5. CRISPR/Cas9 and the Paradigm Shift in Mouse Genome Manipulation Technologies

   • Channabasavaiah B. Gurumurthy, Rolen M. Quadros, Masahiro Sato, Tomoji Mashimo, K. C. Kent Lloyd, Masato Ohtsuka

   Pages 65-77

6. Genome-Editing Technology in CRISPR/Cas System: How to Increase Knock-In Efficiency in Mouse Zygotes

   • Takuro Horii, Izuho Hatada

   Pages 79-91

7. Developments in the Generation of Reporter Stem Cells

   • Samvel Avagyan, Kyle Sylakowski, Uma Lakshmipathy

   Pages 93-106

8. Current Status of Genome Editing in Cardiovascular Medicine

   • Vittavat Termglinchan, Ioannis Karakikes, Timon Seeger, Joseph C. Wu

   Pages 107-126

9. Using CRISPR-Cas9 Genome Editing to Enhance Cell Based Therapies for the Treatment of Diabetes Mellitus

   • Nicole A. J. Krentz, Francis C. Lynn

   Pages 127-147

10. Genome Editing in the Retina: A Case Study in CRISPR for a Patient-Specific Autosomal Dominant Retinitis Pigmentosa Model

    • Sally Justus, Andrew Zheng, Yi-Ting Tsai, Wen-Hsuan Wu, Chun-Wei Hsu, Wei-Pu Wu et al.

    Pages 149-162

11. Back Matter

    Pages 163-166

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This timely volume explores the use of CRISPR-Cas9 for genome editing, presenting cutting-edge techniques and their applications in treatment of disease. The chapters describe latest methods such as use of targetable nucleases, investigation of the non-coding genome, mouse genome editing, increasing of knock-in efficiency in mouse zygotes, and generation of reporter stem cells; the text contextualizes these methods in treatment of cardiovascular disease, diabetes mellitus, retinitis pigmentosa, and others. The final chapters round out the book with a discussion of controversies and future directions.


Genome Editing is an essential, of-the-moment contribution to this rapidly growing field. Drawing from a wealth of international perspectives, it presents novel techniques and applications for the engineering of the human genome. This book is essential reading for all clinicians and researchers in stem cells, regenerative medicine, genomics, biochemicaland biomedical engineering- especially those interested in learning more about genome editing and applying it in a targeted, specific way.

• CRISPR
• ESC
• iPSC
• nucleases
• stem cells

• Ottawa, Canada

  Kursad Turksen

Kursad Turksen received his Ph.D. from the University of Toronto in Toronto, Canada in the area of osteoprogenitor biology and cell selection methodologies. He completed his postdoctoral training at the Howard Hughes Medical Institute, University of Chicago, in Chicago, Illinois, studying epidermal biology through use of genetically-altered mouse models. He joined the Ottawa Hospital Research Institute in Ottawa, Canada [previously known as the Loeb Research Institute] where he rose to the position of Senior Scientist in the Division of Regenerative Medicine. His research interests focus on stem cell biology, with a particular interest in the Claudin family of tight junction proteins and their role in epidermal lineage commitment and progression during development in health and disease. Dr. Turksen is currently Editor in Chief of the Springer journal Stem Cell Reviews and Reports in addition to serving as series editor for the Springer series Stem Cell Biology and Regenerative Medicine.

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