Table of Contents

Preface
Gianluca Petris
1. Making sense of heritable human genome editing: scientific and ethical considerations
Andy Greenfiel
2. CRISPR genome engineering for retinal diseases
Ariel Kantor, Michelle E. McClements, Caroline F. Peddle, Lewis E. Fry, Ahmed Salman, Jasmina Cehajic-Kapetanovic, Kanmin Xue and Robert E. MacLaren
3. Advances in gene editing strategies for epidermolysis bullosa
Thomas Kocher and Ulrich Koller
4. Targeted genome editing for the correction or alleviation of primary immunodeficiencies
Christopher J. Sipe, Patricia N. Claudio Vázquez, Joseph G. Skeate, R. Scott McIvor and Branden S. Moriarity
5. Genome editing approaches to β-hemoglobinopathies
Mégane Brusson and Annarita Miccio
6. Rewriting CFTR to cure Cystic Fibrosis
Giulia Maule, Marjolein Ensinck, Mattijs Bulcaen and Marianne S. Carlon
7. Gene editing and modulation for Duchenne muscular dystrophy
Anthony A. Stephenson and Kevin M. Flanigan
8. Genome editing in the human liver: progress and translational considerations
Samantha L. Ginn, Sharntie Christina and Ian E. Alexander
9. Genome editing in lysosomal disorders
Luisa Natalia Pimentel-Vera, Edina Poletto, Esteban Alberto Gonzalez, Fabiano de Oliveira Poswar, Roberto Giugliani and Guilherme Baldo
10. Genome editing in mucopolysaccharidoses and mucolipidoses
Hallana Souza Santos, Edina Poletto, Roselena Schuh, Ursula Matte and Guilherme Baldo
11. Gene and epigenetic editing in the treatment of primary ciliopathies
Elisa Molinari and John A. Sayer
12. Genome editing in stem cells for genetic neurodisorders
Dell'Amico Claudia, Tata Alice, Pellegrino Enrica, Onorati Marco and Conti Luciano
13. Reprogramming translation for gene therapy
Chiara Ambrosini, Francesca Garilli and Alessandro Quattrone
14. Synthetic genomics for curing genetic diseases
Simona Grazioli and Gianluca Petris

About the Author

Gianluca Petris is currently a Marie Skłodowska-Curie European Fellow and Investigator Scientist developing synthetic genomics tools at the Medical Research Council (MRC) Laboratory of Molecular Biology, Cambridge, United Kingdom. Prior to that, as a postdoctoral fellow at the Department of Cellular, Computational and Integrative Biology (CIBIO), University of Trento, Italy, he developed and applied CRISPR technologies and delivery vehicles for the correction of genetic diseases. Dr. Petris published several papers in the field of CRISPR-therapeutics and CRISPR applications, filed four patent families and co-founded a company for the development of gene therapies for genetic diseases. He was awarded a MSc Degree in Medical Biotechnology working on antibody engineering and rotavirus at the University of Trieste, Italy, and a PhD in Life Sciences (Open University, United Kingdom) in recognition of his research on protein folding and protein quality control carried out at the International Centre for Genetic Engineering and Biotechnology (ICGEB), Trieste, Italy.