Mechanisms of mitochondrial toxicity in Snyder-Robinson Syndrome

Awardee: Grace Zhai

Institution: University of Miami Miller School of Medicine

Award Amount: $42,866

Funding Period: January 1, 2018 - December 31, 2018

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Correction of splice mutations in Ataxia-Telangiectasia through novel antisense oligonucleotides

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Brain MRI signatures in infants with infantile forms of GM-1 and GM-2