Seeking cures : design of therapies for genetically determined diseases / Moyra Smith, MD, PhD, MFA.

Oxford ; New York : Oxford University Press, [2014]

xi, 296 pages : illustrations ; 25 cm

Includes bibliographical references (pages 241-282) and index.

Introduction and history -- Therapy : design and general approaches -- Inborn errors of metabolism : progress in diagnosis and treatment -- Lysosomal storage diseases and therapies -- Mitochondrial function, defects and approaches to treatment -- Protein misfolding, endoplasmic reticulum stress and pathogenesis of disease -- Transporters and solute carriers : passage of molecules across membranes -- Advances in therapy for monogenic diseases -- Identifying therapeutic targets in complex, multifactorial diseases -- Approaches to cancer treatment -- Gene based molecular therapies -- Stem cells and pluripotent stem cells.