03138nam 2200601Ia 450 991077985690332120230803220021.00-19-021309-40-19-991587-3(CKB)2550000001106564(EBL)1336510(OCoLC)855504970(SSID)ssj0000950739(PQKBManifestationID)12421535(PQKBTitleCode)TC0000950739(PQKBWorkID)10881627(PQKB)11338547(StDuBDS)EDZ0001535417(MiAaPQ)EBC1336510(StDuBDS)EDZ0000864019(Au-PeEL)EBL1336510(CaPaEBR)ebr10740048(CaONFJC)MIL506929(EXLCZ)99255000000110656420130205d2014 uy 0engur|n|---|||||txtccrSeeking cures[electronic resource] design of therapies for genetically determined diseases /Moyra SmithOxford ;New York Oxford University Pressc20141 online resource (311 p.)Description based upon print version of record.0-19-991586-5 1-299-75678-6 Includes bibliographical references and index.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 Diseases10. 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; ZProgress 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 heGenetic disordersGene therapyGenetic disorders.Gene therapy.616/.042Smith Moyra1130727MiAaPQMiAaPQMiAaPQBOOK9910779856903321Seeking cures3736170UNINA