Chris Lorson, associate vice chancellor for research and strategic initiatives
Christian Lorson wants to give hope to families who have dealt with incurable genetic diseases for generations.
His research has largely focused on spinal muscular atrophy, a degenerative disease that affects one in 6,000 live births. Through the NextGen Precision Health initiative, Lorson is taking his knowledge of the disease and applying it to another incurable genetic diseases like Charcot-Marie Tooth (CMT).
CMT progressively deteriorates every nerve outside of the brain and spinal cord. Even though the disease currently has no cure, Lorson said it’s primed for therapeutic intervention through precision medicine. “Like much of the research at NextGen, the goal is to develop a treatment that will be delivered to a specific person — and their specific cells,” he said.
Lorson’s colleagues say he’s the perfect person for the job.
“Christian is fantastic at making viruses to deliver therapeutic molecules,” said Michael L. Garcia, associate professor of biological sciences at the University of Missouri. “One of the big conundrums of diseases that affect the nervous system, in particular, is that you're born with the mutation. If we can actually change the way the cell's DNA is working, we can make a bad gene work correctly.”