TY - CHAP M1 - Book, Section TI - Genetic Therapeutics A1 - Schaefer, G. Bradley A1 - Thompson, Jr., James N. Y1 - 2017 N1 - T2 - Medical Genetics: An Integrated Approach AB - By this point in the book, we feel sure you as readers have appreciated the heavy emphasis on diagnostics. In fact, the mainstay of clinical genetics is still in identifying the etiology and pathogenesis of specific disorders. However, as the practice of clinical genetics has progressed, so have advances in therapies. There are now several available modalities of genetic treatments. Up until recently, however, most treatments provided by medical geneticists primarily involved counseling and case management. The treatment of inborn errors of metabolism (IEM) dates back to the mid-1960s. These treatments have involved dietary adjustments, specialized formulas, and vitamin/cofactor supplementation. More recently tissue transplantation and enzyme replacement therapies have become available. New treatment modalities have been developed for non-metabolic disorders. Bioengineered pharmaceuticals are now commonplace. Other treatment options like personalized medicine strategies, tissue cloning, gene correction, and true gene therapy all sit poised for transition out of clinical trials and into standard medical care. In the first section of this chapter we will discuss the mechanisms of the different modes of genetic therapies. In the second section we will discuss the clinical application of these therapies. SN - PB - McGraw-Hill Education CY - New York, NY Y2 - 2024/03/29 UR - accessmedicine.mhmedical.com/content.aspx?aid=1147723440 ER -