Study in mice shows genes may be altered through drug repurposing
Researchers at the University of Illinois Chicago have published a study showing a promising approach to using drug repurposing to treat genetic diseases.
A team from the UIC Department of Ophthalmology and Visual Sciences published the article, Gene dosage manipulation alleviates manifestations of hereditary PAX6 haploinsufficiency in mice in the journal Science Translational Medicine.
Nearly all the genes in human DNA have two copies, one inherited from the mother and one from the father. There are some genetic diseases where only one copy is normal and the other one is non-functional due to a mistake in the DNA. The idea behind this study was to see if the normal copy can be enhanced to make up for the non-functional copy, said Ali Djalilian, UIC professor of ophthalmology and corresponding author of the paper.