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Patient stem cells used to make dementia-in-a-dish; help identify new treatment strategy

Belgian researchers have identified a new strategy for treating an inherited form of after attempting to turn derived from patients into the neurons most affected by the disease. In patient-derived carrying a mutation predisposing them to , which accounts for about half of dementia cases before the age of 60, the scientists found a targetable defect that prevents normal neurodevelopment. These partially return to normal when the defect is corrected.

iPSCs Derived from Patients with Frontotemporal Dementia
Induced (iPSCs) derived from patients with frontotemporal dementia were genetically corrected and converted to . The green staining indicates the cortical marker CTIP2, the red stain is the neuronal marker TUJ1, and the blue stains the nuclei of the cells.
Image credit: Susanna Raitano/Stem Cell Reports 2014


Stem Cell Reports, Raitano et al. Restoration of Progranulin Expression Rescues Cortical Neuron Generation in an Induced Pluripotent Stem Cell Model of Frontotemporal Dementia

Cell Press