Targeting miR-155 as a therapy for mucolipidosis IV

Awardee: Susan Slaugenhaupt

Institution: Massachusetts General Hospital

Award Amount: $50,000

Funding Period: January 1, 2015 - December 31, 2015

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Unraveling the basis of white matter disease in mucopolysaccharidosis type I – a DTI study

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Identification of Molecular Targets for Pitt-Hopkins Syndrome Treatments