Gene Therapy Restores Dopamine Transporter Expression and Ameliorates Pathology in iPSC and Mouse Models of Infantile Parkinsonism

Using patient-derived iPSCs, researchers generated a midbrain dopaminergic neuron model of dopamine transporter deficiency syndrome that exhibited marked impairment of DAT activity, apoptotic neurodegeneration associated with TNFα-mediated inflammation, and dopamine toxicity.