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Author:   Moyra Smith, MD, PhD (Professor Emeritus, Department of Pediatrics, University of California at Irvine, Irvine, California, USA)
Publisher:   Oxford University Press Inc
Imprint:   Oxford University Press Inc
Dimensions:   Width: 23.60cm , Height: 2.00cm , Length: 16.30cm
Weight:   0.587kg
ISBN:  

9780199915866

ISBN 10:   0199915865
Pages:   320
Publication Date:   19 September 2013
Audience:   College/higher education ,  Professional and scholarly ,  Postgraduate, Research & Scholarly ,  Professional & Vocational
Format:   Hardback
Publisher's Status:   Active
Availability:   Available To Order  
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Table of Contents

Preface Chapter 1: Introduction and History Chapter 2: Therapy: design and general approaches Chapter 3: Inborn errors of metabolism: progress in diagnosis and treatment Chapter 4: Lysosomal storage diseases and therapies Chapter 5: Mitochondrial function, defects and approaches to treatment Chapter 6: Protein misfolding, endoplasmic reticulum stress and pathogenesis of disease Chapter 7: Transporters and solute carriers: passage of molecules across membranes Chapter 8: Advances in therapy for monogenic diseases Chapter 9: Identifying therapeutic targets in complex, multifactorial diseases Chapter 10: Approaches to cancer treatment Chapter 11: Gene based molecular therapies Chapter 12; Stem cells and pluripotent stem cells Epilogue (Envoi) References

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Author Information

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 genomics in autism.

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