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Record Nr. |
UNINA9910779856903321 |
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Autore |
Smith Moyra |
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Titolo |
Seeking cures [[electronic resource] ] : design of therapies for genetically determined diseases / / Moyra Smith |
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Pubbl/distr/stampa |
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Oxford ; ; New York, : Oxford University Press, c2014 |
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ISBN |
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0-19-021309-4 |
0-19-991587-3 |
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Descrizione fisica |
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1 online resource (311 p.) |
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Disciplina |
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Soggetti |
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Genetic disorders |
Gene therapy |
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Lingua di pubblicazione |
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Formato |
Materiale a stampa |
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Livello bibliografico |
Monografia |
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Note generali |
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Description based upon print version of record. |
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Nota di bibliografia |
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Includes bibliographical references and index. |
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Nota di contenuto |
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Cover; Contents; Preface; Acknowledgments; 1. Introduction and History; 2. Therapy: Design and General Approaches; 3. Inborn Errors of Metabolism: Progress in Diagnosis and Treatment; 4. Lysosomal Storage Diseases and Therapies; 5. Mitochondrial Function, Defects, and Approaches to Treatment; 6. Protein Misfolding, Endoplasmic Reticulum Stress, and Pathogenesis of Disease; 7. Transporters and Solute Carriers: Proteins That Transport Molecules Across Membranes; 8. Advances in Therapy for Specific Monogenic Diseases; 9. Identifying Therapeutic Targets in Complex, Multifactorial Diseases |
10. Approaches to Cancer Treatment11. Gene-Based Molecular Therapies; 12. Stem Cells and Induced Pluripotent Stem Cells; Epilogue (Envoi); References; Index; A; B; C; D; E; F; G; H; I; J; K; L; M; N; O; P; Q; R; S; T; U; V; W; X; Y; Z |
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Sommario/riassunto |
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Progress in molecular and cellular biology has greatly enhanced our ability to accurately diagnose diseases that are caused by gene mutations, changes in genome structures, and altered gene expression; increased emphasis is now placed on translational research and the clinical treatment of these genetically determined diseases. Seeking Cures outlines the progress and implications of science's quest to identify therapeutic targets and initiate novel treatments at the gene, RNA, protein, and physiological levels. Also considered are aspects of |
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