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Implanting Genetically Corrected Stem Cells Partially Restores Enzyme Activity in Sanfilippo Mice, Study Finds

Brain transplantation of genetically corrected stem cells partially restored enzymatic activity in mice with Sanfilippo syndrome, according to UCLA researchers. Their study, “Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice,” was published in the journal Molecular Therapy Methods…

Restoring Activity of NAGLU Enzyme Corrected Metabolic Abnormalities in Sanfilippo, Mouse Study Finds

Restoring the activity of the alpha-N-acetylglucosaminidase (NAGLU) enzyme using an artificial viral vector successfully corrected metabolic abnormalities in a mouse model of Mucopolysaccharidosis IIIB (MPS IIIB), also known as Sanfilippo syndrome, a new study using mice shows. The study, “Near-Complete Correction of Profound Metabolomic Impairments Corresponding to Functional…