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Germline biallelic mutations in\n                    <jats:italic>WWOX<\/jats:italic>\n                    cause WWOX\u2010related 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 epileptic encephalopathy and profound global developmental delay. We recently demonstrated that neuronal ablation of murine\n                    <jats:italic>Wwox<\/jats:italic>\n                    recapitulates phenotypes of\n                    <jats:italic>Wwox<\/jats:italic>\n                    \u2010null mice leading to intractable epilepsy, hypomyelination, and postnatal lethality. Here, we designed and produced an adeno\u2010associated viral vector (AAV9) harboring murine\n                    <jats:italic>Wwox<\/jats:italic>\n                    or human\n                    <jats:italic>WWOX<\/jats:italic>\n                    cDNA and driven by the human neuronal Synapsin I promoter (\n                    <jats:italic>AAV\u2010SynI\u2010WWOX<\/jats:italic>\n                    ). Testing the efficacy of AAV\u2010SynI\u2010WWOX delivery in\n                    <jats:italic>Wwox<\/jats:italic>\n                    \u2010null mice demonstrated that specific neuronal restoration of WWOX expression rescued brain hyperexcitability and seizures, hypoglycemia, myelination deficits, and the premature lethality and behavioral deficits of\n                    <jats:italic>Wwox<\/jats:italic>\n                    \u2010null mice. These findings provide a proof\u2010of\u2010concept for\n                    <jats:italic>WWOX<\/jats:italic>\n                    gene therapy as a promising approach to curing children with WOREE and SCAR12.\n                  <\/jats:p>","DOI":"10.15252\/emmm.202114599","type":"journal-article","created":{"date-parts":[[2021,11,8]],"date-time":"2021-11-08T02:52:36Z","timestamp":1636339956000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":22,"title":["Neonatal neuronal WWOX gene therapy rescues Wwox null phenotypes"],"prefix":"10.1038","volume":"13","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-1654-5710","authenticated-orcid":false,"given":"Srinivasarao","family":"Repudi","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irina","family":"Kustanovich","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sara","family":"Abu\u2010Swai","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2644-7068","authenticated-orcid":false,"given":"Shani","family":"Stern","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6034-023X","authenticated-orcid":false,"given":"Rami I","family":"Aqeilan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2021,11,7]]},"reference":[{"key":"BFEMMM202114599_CR1","doi-asserted-by":"crossref","first-page":"1177","DOI":"10.1080\/15384101.2019.1616998","volume":"18","author":"SK Abdeen","year":"2019","unstructured":"Abdeen SK, Aqeilan RI (2019) Decoding the link between WWOX and p53 in aggressive breast cancer. 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