Article
Recombinant Adeno-Associated Virus-mediated rescue of function in a mouse model of Dopamine Transporter Deficiency Syndrome.
Scientific reports - 18 Apr 2017
Illiano P, Bass C E, Fichera L, Mus L, Budygin E A, Sotnikova T D, Leo D, Espinoza S, Gainetdinov R R
Abstract excerpt
Dopamine Transporter Deficiency Syndrome (DTDS) is a rare autosomal recessive disorder caused by loss-of-function mutations in dopamine transporter (DAT) gene, leading to severe neurological disabilities in children and adults. DAT-Knockout (DAT-KO) mouse is currently the best animal model for this syndrome, displaying functional hyperdopaminergia and neurodegenerative phenotype leading to premature death in ~36%...
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