Gene Therapy for Oculopharyngeal Muscular Dystrophy
The PABPN1 Gene
This gene is responsible for making a protein that will be active in the entirety of the body. It plays an important role in the processing of mRNA which serve as a blueprint for making proteins. In its capacity it binds to the end of mRNA molecule on the poly (A) tail which is composed of adenine molecules. The poly (A) tails are necessary in preventing mRNA from breaking down as they move inside of a cell. PABPN1 adds adenines to the poly (A) through polyadenylation and may also be involved in the regulation of mRNA production.
