Seeking Cures: Design of Therapies for Genetically Determined Diseases

Hardcover | September 24, 2013

byMoyra Smith

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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 the clinical treatment of thesegenetically 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 orinduced pluripotent stem cells). Topics covered in this text include: * outline of the processes typical for identifying disease-modifying therapies* examples of newer therapeutic approaches in use or under investigation to treat lysosomal storage diseases, inborn errors of metabolism, mitochondrial functional defects, and specific monogenic diseases * therapeutic designs for specific complex common diseases, including Alzheimer's disease, cancer, and autismThrough these specific examples, Seeking Cures provides a glimpse at the pursuit - and future - of personalized medicine.

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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 the clinical treatment of thesegenetically determined diseases. Seeking Cure...

Moyra Smith, MD, PhD, MFA, has been conductiong research in human genetics since 1965. For 19 years she served as Clinical Geneticist and Director of the Newborn Screening Program at the University of California, Irvine, where she now serves as Professor Emeritus. Her latest research includes studies of nuclear and mitochondiral genomi...

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Format:HardcoverDimensions:304 pages, 9.25 × 6.12 × 0.98 inPublished:September 24, 2013Publisher:Oxford University PressLanguage:English

The following ISBNs are associated with this title:

ISBN - 10:0199915865

ISBN - 13:9780199915866

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Table of Contents

Preface1. Introduction and History2. Therapy: design and general approaches3. Inborn errors of metabolism: progress in diagnosis and treatment4. Lysosomal storage diseases and therapies5. Mitochondrial function, defects and approaches to treatment6. Protein misfolding, endoplasmic reticulum stress and pathogenesis of disease7. Transporters and solute carriers: passage of molecules across membranes8. Advances in therapy for monogenic diseases9. Identifying therapeutic targets in complex, multifactorial diseases10. Approaches to cancer treatment11. Gene based molecular therapies12. Stem cells and pluripotent stem cellsEpilogue (Envoi)References