Towards Precision Medicine in the Treatment of Congenital Hyperinsulinism in Infancy

Awardee: Mark Dunne

Institution: University of Manchester

Award Amount: $71,000

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

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Optimizing Delivery and Endpoints of an AAV9-mediated Gene Therapy Trial in AMN

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Systematic Evaluation of a Cluster of Nonsense Mutations in Exon 22 of CFTR to Inform Treatment