Curing Genetic Diseases through Genome Reprogramming
Gebonden Engels 2021 9780323853019Levertijd ongeveer 9 werkdagen
Gratis verzonden
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.
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Inhoudsopgave
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 β-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
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 β-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
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