Originally reported by Columbia Daily Tribune, researchers from the University of Missouri have made huge strides towards treating, and maybe even curing, two fatal diseases: muscular dystrophy and Amyotrophic Lateral Sclerosis (ALS). Dongsheng Duan and Singhua Ding, are on their way towards medical greatness, but first, successful clinical human trials have to happen.
Duan, at MU’s School of Medicine, successfully used gene therapy on dogs to prevent muscular dystrophy back in 2015. Muscular dystrophy is an aggressive disease that disrupts protein dystrophin production, leading to deteriorated muscles. As the disease progresses, it will eventually attack all muscles, including the lungs and the heart, which has fatal consequences. However Duan and his team were able to develop a microgene referred to as microdystrophin (SGT-001) and use that to replace the mutated gene. The trials are set to take place soon for human testing soon in the US. They will ensure sure it’s effective and safe.
Meanwhile, Ding remains at Dalton Cardiovascular experimenting for ALS treatment and eventually a cure. ALS, also referred to as Lou Gehrig’s disease, is a also a degenerative muscular disease, but it stems from motor neurons. Ding and his team are focusing on a NAMPT enzyme (which remains in the brain) to hopefully delay the onset of muscle decay. After testing it among mice, he found that mice with the enzyme experienced several symptoms found in ALS and they were able to successfully restore their function after treatment with nicotinamide mononucleotide. The communications director at the Mid-America Chapter of the ALS Association is very impressed with the latest research and encouraged them to apply to receive a grant through their organization.
These hardworking researchers and their teams are so close to what could be two gigantic medical breakthroughs. It’s only a matter of time until they conduct human trials and hopefully they find success to help aid thousands upon thousands affected from these two fatal diseases.