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Down Syndrome
Research from J. Ortizabalia and co-researchers provides new data on Down syndrome
December 18th, 2008
"Genetic-dissection studies carried out with Down syndrome (DS) murine models point to the critical contribution of Dyrk] A overexpression to the motor abnormalities and cognitive deficits displayed in DS individuals. In the present study we have used a murine model overexpressing Dyrk1A (TgDyrk1A mice) to evaluate whether functional CNS defects could be corrected with an inhibitory RNA against Dyrk]A, delivered by bilateral intrastriatal injections of adeno-associated virus type 2 (AAVshDyrk1A)," investigators in Barcelona, Spain report. "We report that AAVshDyrk1A efficiently transduced HEK293 cells and primary neuronal cultures, triggering the specific inhibition of...
Source: Gene Therapy Weekly (2008-12-18)
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