Subbotina E, Koganti S R K, Hodgson-Zingman D M, Zingman L V
Department of Internal Medicine, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA.
Fraternal Order of Eagles Diabetes Research Center, University of Iowa, Iowa City, Iowa, USA.
Clin Pharmacol Ther. 2016 Jan;99(1):21-5. doi: 10.1002/cpt.276. Epub 2015 Nov 10.
The development of genetic and molecular biology tools permitting the connection of specific genes to their functions has accelerated our understanding of molecular pathways underlying health and disease. The resulting gains in knowledge have propelled gene targeting to the forefront of promising therapeutic strategies. Here we discuss the uniquely powerful and adaptable approach of morpholino-driven modification of normal and mutant gene expression as a pathway to health.