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).
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).
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.
Inhaltsangabe
* 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
