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Thrilled to see our mouse gene therapy for Dopamine Transporter Deficiency Syndrome. DTDS is a rare inherited genetic disease, caused by mutated SLC6A3 encoding dopamine transporter. stm.sciencemag.org/content/13/594…
Dopamine transporter reuptakes dopamine from the synaptic cleft after neurotransmission. Current treatments are palliative, not very effective, and prognosis is very poor.
For me, the story started 21/9/2011. @profahadrahim, @drsuzybuckley +me were extending our #genetherapy to harder targets such as neuronopathic #GaucherDisease @gauchersA. Paediatricians Paul Gissen & Manju Kurian () () emailed.
