Article

<i>Juvenile</i> AAV-Mediated MEF2C Gene Replacement Ameliorates Selected Phenotypes in <i>Mef2c</i> -Haploinsufficient Mice

2026-08-18

Abstract excerpt

MEF2C haploinsufficiency syndrome is a severe neurodevelopmental disorder for which no disease-directed treatment is available. We investigated whether neuron-directed adeno- associated virus (AAV) delivery of a functional MEF2C coding sequence during the juvenile period could modify disease-relevant phenotypes in mice heterozygous for a Mef2c exon 4 deletion. Transcript-level analysis identified a brain-enriched...

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Literature Corpus work: ce077816-bd7f-515e-83cb-624ba26fc4fc
DOI: 10.64898/2026.08.14.744746

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<i>Juvenile</i> AAV-Mediated MEF2C Gene Replacement Ameliorates Selected Phenotypes in <i>Mef2c</i> -Haploinsufficient MiceDOI 10.64898/2026.08.14.744746Read

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