Stem Cells From MPSIIIA Patient Provide Potentially Useful Tool for Disease Modeling and Therapy
For the first time, researchers have created and validated two lines of stem cells derived from the skin cells of a patient with mucoplysaccharydosis IIIA (MPSIIIA), the most severe form of Sanfilippo syndrome. According to the scientists, these patient-derived stem cells could be a useful tool not only to study the…