Recombinant Adeno-Associated Virus-mediated rescue of function in a mouse model of Dopamine Transporter Deficiency Syndrome.
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ABSTRACT:
SUBMITTER: Illiano P
PROVIDER: S-EPMC5394687 | biostudies-literature | 2017 Apr
REPOSITORIES: biostudies-literature
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Recombinant Adeno-Associated Virus-mediated rescue of function in a mouse model of Dopamine Transporter Deficiency Syndrome.
Illiano P P Bass C E CE Fichera L L Mus L L Budygin E A EA Sotnikova T D TD Leo D D Espinoza S S Gainetdinov R R RR
Scientific reports 20170418
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% of the population. We used DAT-KO mouse as model for DTDS to explore the potential ...[more]
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