New DNA Treatment Shows Promise in Delaying ALS Paralysis.
A team of scientists led by Don Cleveland at the University of California San Diego School of Medicine has developed a designer DNA drug that can restore normal levels of stathmin-2, a protein crucial to the regeneration of neurons and the maintenance of their connections to muscle fibers, which is lost in almost all persons with amyotrophic lateral sclerosis (ALS). The drug works by mimicking the function of TDP-43, a protein associated with ALS, Alzheimer's disease, and other neurodegenerative disorders. The findings lay the foundation for a clinical trial to delay paralysis in ALS by maintaining stathmin-2 protein levels in patients using the designer DNA drug.