Curing Genetic Diseases through Genome Reprogramming

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.

• PrefaceGianluca Petris1. Making sense of heritable human genome editing: scientific and ethical considerationsAndy Greenfiel2. CRISPR genome engineering for retinal diseasesAriel Kantor, Michelle E. McClements, Caroline F. Peddle, Lewis E. Fry, Ahmed Salman, Jasmina Cehajic-Kapetanovic, Kanmin Xue and Robert E. MacLaren3. Advances in gene editing strategies for epidermolysis bullosaThomas Kocher and Ulrich Koller4. Targeted genome editing for the correction or alleviation of primary immunodeficienciesChristopher J. Sipe, Patricia N. Claudio Vázquez, Joseph G. Skeate, R. Scott McIvor and Branden S. Moriarity5. Genome editing approaches to β-hemoglobinopathiesMégane Brusson and Annarita Miccio6. Rewriting CFTR to cure Cystic FibrosisGiulia Maule, Marjolein Ensinck, Mattijs Bulcaen and Marianne S. Carlon7. Gene editing and modulation for Duchenne muscular dystrophyAnthony A. Stephenson and Kevin M. Flanigan8. Genome editing in the human liver: progress and translational considerationsSamantha L. Ginn, Sharntie Christina and Ian E. Alexander9. Genome editing in lysosomal disordersLuisa Natalia Pimentel-Vera, Edina Poletto, Esteban Alberto Gonzalez, Fabiano de Oliveira Poswar, Roberto Giugliani and Guilherme Baldo10. Genome editing in mucopolysaccharidoses and mucolipidosesHallana Souza Santos, Edina Poletto, Roselena Schuh, Ursula Matte and Guilherme Baldo11. Gene and epigenetic editing in the treatment of primary ciliopathiesElisa Molinari and John A. Sayer12. Genome editing in stem cells for genetic neurodisordersDell'Amico Claudia, Tata Alice, Pellegrino Enrica, Onorati Marco and Conti Luciano13. Reprogramming translation for gene therapyChiara Ambrosini, Francesca Garilli and Alessandro Quattrone14. Synthetic genomics for curing genetic diseasesSimona Grazioli and Gianluca Petris