| Description |
1 online resource |
| Contents |
Introduction and history -- Therapy : design and general approaches -- Inborn errors of metabolism : progress in diagnosis and treatment -- Lysosomal storage diseases and therapies -- Mitochondrial function, defects and approaches to treatment -- Protein misfolding, endoplasmic reticulum stress and pathogenesis of disease -- Transporters and solute carriers : passage of molecules across membranes -- Advances in therapy for monogenic diseases -- Identifying therapeutic targets in complex, multifactorial diseases -- Approaches to cancer treatment -- Gene based molecular therapies -- Stem cells and pluripotent stem cells |
| Summary |
"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 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 treatment at the cellular level (e.g., those with hematopoietic stem cells or induced pluripotent stem cells)"--Publisher's description |
| Bibliography |
Includes bibliographical references and index |
| Notes |
English |
|
Print version record |
| Subject |
Genetic disorders -- Diagnosis.
|
|
Genetic disorders -- Treatment
|
|
Gene therapy.
|
|
Genetic Diseases, Inborn -- therapy
|
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Genetic Therapy
|
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HEALTH & FITNESS -- Diseases -- Genetic.
|
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MEDICAL -- Genetics.
|
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Gene therapy
|
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Genetic disorders -- Diagnosis
|
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Genetic disorders -- Treatment
|
| Form |
Electronic book
|
| LC no. |
2013004975 |
| ISBN |
9780199915873 |
|
0199915873 |
|
9780199326051 |
|
0199326053 |
|
0190213094 |
|
9780190213091 |
|
