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100   $a20130205d2014      uy             0
101 0 $aeng
135   $aur|n|---|||||
181   $ctxt
182   $cc
183   $acr
200 10$aSeeking cures$b[electronic resource] $edesign of therapies for genetically determined diseases /$fMoyra Smith
210   $aOxford ;$aNew York $cOxford University Press$dc2014
215   $a1 online resource (311 p.)
300   $aDescription based upon print version of record.
311   $a0-19-991586-5 
311   $a1-299-75678-6 
320   $aIncludes bibliographical references and index.
327   $aCover; 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
327   $a10. 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
330   $aProgress 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 treatment at the cellular level (e.g., those with he
606   $aGenetic disorders
606   $aGene therapy
608   $aElectronic books.
615  0$aGenetic disorders.
615  0$aGene therapy.
676   $a616/.042
700   $aSmith$b Moyra$0903961
801  0$bMiAaPQ
801  1$bMiAaPQ
801  2$bMiAaPQ
906   $aBOOK
912   $a9910452945503321
996   $aSeeking cures$92189486
997   $aUNINA