Curing Genetic Diseases through Genome Reprogramming
Specificaties
Gebonden, blz.
|
Engels
Elsevier Science |
e druk, 2021
ISBN13: 9780323853019
Rubricering
Onderdeel van serie
Progress in Molecular Biology and Translational Science
Levertijd ongeveer 8 werkdagen
Samenvatting
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.
Specificaties
Inhoudsopgave
<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>