Featured Profile: Chris Lorson
University of Missouri geneticist Dr. Chris Lorson is combating childhood neurodegenerative disease.
Spinal muscular atrophy (SMA) is a devastating neurodegenerative disease and is the leading genetic cause of infantile death worldwide. Children with SMA lose muscle tissue over time, eventually becoming wheelchair bound or succumbing to respiratory complications.
Lorson, who has been researching SMA for 17 years, has developed a new compound that proved highly effective in treating mice with the disease. For a healthy mouse, life expectancy is two years; mice with SMA live an average of two weeks. When Lorson treated sick mice with the compound, they lived 10-12 times longer.
Lorson also demonstrated that the earlier treatment begins, the better the outcome. He hopes one day newborns will be routinely screened to diagnose and treat SMA before symptoms appear.
Although there is currently no cure, the National Institutes of Health has listed SMA as the neurological disease closest to being cured, due in part to effective drugs like the one being developed in Lorson’s lab.
Dr. Chris Lorson is a professor of veterinary pathobiology in the College of Veterinary Medicine, professor of molecular microbiology and immunology in the School of Medicine and a researcher at MU’s Bond Life Sciences Center.
Click here to download a .pdf of this story.
One Health/One Medicine