Evaluating “exon‐skipping” as a strategy to repair the CFTR nonsense mutation

Awardee: Christine Bear

Institution: SickKids Hospital-Toronto

Award Amount: $51,500

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

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CNS Protection from Hyperammonemia

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