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.
1. Cover image - p. i 2. Title page - p. ii 3. Table of Contents - p. iii 4. Copyright - p. iii 5. Contributors - p. iii 6. Preface - p. xvii 7. Chapter One: Making sense of heritable human genome editing: Scientific and ethical considerations - p. 1 8. Chapter Two: CRISPR genome engineering for retinal diseases - p. 66 9. Chapter Three: Advances in gene editing strategies for epidermolysis bullosa - p. 89 10. Chapter Four: Targeted genome editing for the correction or alleviation of primary Immunodeficiencies - p. 111 11. Chapter Five: Genome editing approaches to β-hemoglobinopathies - p. 153 12. Chapter Six: Rewriting CFTR to cure cystic fibrosis - p. 185 13. Chapter Seven: Gene editing and modulation for Duchenne muscular dystrophy - p. 225 14. Chapter Eight: Genome editing in the human liver: Progress and translational considerations - p. 260 15. Chapter Nine: Genome editing in lysosomal disorders - p. 294 16. Chapter Ten: Genome editing in mucopolysaccharidoses and mucolipidoses - p. 327 17. Chapter Eleven: Gene and epigenetic editing in the treatment of primary ciliopathies - p. 360 18. Chapter Twelve: Genome editing in stem cells for genetic neurodisorders - p. 403 19. Chapter Thirteen: Reprogramming translation for gene therapy - p. 439 20. Chapter Fourteen: Synthetic genomics for curing genetic diseases - p. 498 21. Index - p. 520
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.
1. Cover image - p. i 2. Title page - p. ii 3. Table of Contents - p. iii 4. Copyright - p. iii 5. Contributors - p. iii 6. Preface - p. xvii 7. Chapter One: Making sense of heritable human genome editing: Scientific and ethical considerations - p. 1 8. Chapter Two: CRISPR genome engineering for retinal diseases - p. 66 9. Chapter Three: Advances in gene editing strategies for epidermolysis bullosa - p. 89 10. Chapter Four: Targeted genome editing for the correction or alleviation of primary Immunodeficiencies - p. 111 11. Chapter Five: Genome editing approaches to β-hemoglobinopathies - p. 153 12. Chapter Six: Rewriting CFTR to cure cystic fibrosis - p. 185 13. Chapter Seven: Gene editing and modulation for Duchenne muscular dystrophy - p. 225 14. Chapter Eight: Genome editing in the human liver: Progress and translational considerations - p. 260 15. Chapter Nine: Genome editing in lysosomal disorders - p. 294 16. Chapter Ten: Genome editing in mucopolysaccharidoses and mucolipidoses - p. 327 17. Chapter Eleven: Gene and epigenetic editing in the treatment of primary ciliopathies - p. 360 18. Chapter Twelve: Genome editing in stem cells for genetic neurodisorders - p. 403 19. Chapter Thirteen: Reprogramming translation for gene therapy - p. 439 20. Chapter Fourteen: Synthetic genomics for curing genetic diseases - p. 498 21. Index - p. 520
