Motor Neurons in SMA Patients May Express Fewer Key Proteins

Motor neurons in spinal muscular atrophy (SMA) patients express fewer proteins associated with neuronal development than those in healthy individuals, according to a study that used induced pluripotent stem cells from individuals in both groups to create autologous motor neurons. The researchers — led by Heidi Fuller from The Robert Jones and…

Research Finds RNA Therapy Factors Ameliorate SMA Phenotype

In a recent study entitled “Spinal Muscular Atrophy Phenotype Is Ameliorated Either By SMN Increase Or Modulation Of Secondary Cell Death Events With RNA Therapy (S56.006),” a team of researchers from Milan, Italy, using a stem cell model sought to develop treatment approaches based on RNA for patients with…