Seeking CuresDesign of Therapies for Genetically Determined Diseases

Seeking CuresDesign of Therapies for Genetically Determined Diseases

Moyra Smith

Print publication date: Sep 2013

ISBN: 9780199915866

Publisher: Oxford University Press

Abstract

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 include an 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, and therapeutic designs for specific complex common diseases, including Alzheimer's disease, cancer, and autism.