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- 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 Diseases
- 10 Approaches to Cancer Treatment
- 11 Gene-Based Molecular Therapies
- 12 Stem Cells and Induced Pluripotent Stem Cells