Curing Genetic Diseases through Genome Reprogramming

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