Curing Genetic Diseases through Genome Reprogramming, Volume 182 captures an historic moment in the field of gene therapy-the dawn of a new age in which the dream of curing genetic diseases has become realizable. The volume presents the most clinically advanced gene therapy and genome editing approaches for the treatment of genetic diseases in specific organs, including difficult therapeutic targets, futuristic ideas of genetic interventions, and large scale human genome repair. An initial chapter addresses the complex ethical aspects involved in the very idea of modifying the human genome.
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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 V?zquez, Joseph G. Skeate, R. Scott McIvor and Branden S. Moriarity 5. Genome editing approaches to ?-hemoglobinopathies M?gane 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
