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

Awardee: Oded/Anikster Regev/Yair

Institution: New York University

Award Amount: $65,689

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

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Novel alpha-glucosaminidase for Sanfilippo C syndrome

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Mechanisms of mitochondrial toxicity in Snyder-Robinson Syndrome