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AAV9-Mediated Gene Replacement Therapy for CTNNB1-Related Neurodevelopmental Disorder

2026-08-27

Abstract excerpt

<title>Abstract</title> <p> CTNNB1 Syndrome is a severe neurodevelopmental disorder caused by haploinsufficiency of the <italic>CTNNB1</italic> gene, which encodes β-catenin, a central regulatory protein of the canonical Wnt signaling and nervous systems development. Today affected patients experience global developmental delays (GDD), intellectual disability, and progressive motor impairments, with no disease...

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Literature Corpus work: f2c30b71-0c81-5c97-981e-5d087a76af3f
DOI: 10.21203/rs.3.rs-10408032/v1

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