Article

Neonatal neuronal WWOX gene therapy rescues <i>Wwox</i> null phenotypes

2021-04-27

Abstract excerpt

WW domain-containing oxidoreductase ( WWOX ) is an emerging neural gene regulating homeostasis of the central nervous system. Germline biallelic mutations in WWOX cause WWOX-related epileptic encephalopathy (WOREE) syndrome and spinocerebellar ataxia, and autosomal recessive 12 (SCAR12), two devastating neurodevelopmental disorders with highly heterogenous clinical outcomes, the most common being severe epilepti...

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Literature Corpus work: c062bcf3-938a-58b7-b382-3021f79d6099
DOI: 10.1101/2021.04.27.441575

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