Curing Genetic Diseases through Genome Reprogramming

Gianluca Petris is currently a Marie Sklodowska-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.

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 ß-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