Mechanisms of Neurodevelopmental Defects in Mucolipidosis type IV (MLIV)

Awardee: Kartik Venkatachalam

Institution: University of Texas, School of Medicine at Houston

Award Amount: $53,000

Funding Period: January 1, 2016 - December 31, 2016

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Targeted AAV gene therapy for vertical supranuclear gaze palsy and dysphagia

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Regulation of TCF4 transcriptional activity in neurons