Seeking Cures: Design of Therapies for Genetically Determined Diseases

ISBN : 9780199915866

Moyra Smith
320 Pages
163 x 241 mm
Pub date
Sep 2013
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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 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). 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 autism Through these specific examples, Seeking Cures provides a glimpse at the pursuit-and future-of personalized medicine.


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)

About the author: 

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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