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adult onset sandhoff disease presenting with a motor neuron disease phenotype clinical and mechanistic insights from patient derived models
Original title: “Adult-onset Sandhoff disease presenting with a motor neuron disease phenotype: clinical and mechanistic insights from patient-derived models.”
generation and characterization of induced pluripotent stem cell ipsc lines from patients affected with tay sachs and sandhoff disease
Original title: “Generation and characterization of induced pluripotent stem cell (iPSC) lines from patients affected with Tay-Sachs and Sandhoff disease.”