AAV delivery of full-length SYNGAP1 rescues epileptic and behavioral phenotypes in a mouse model of SYNGAP1-related disorders.

doi:10.1016/j.ymthe.2025.09.040

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Article

AAV delivery of full-length SYNGAP1 rescues epileptic and behavioral phenotypes in a mouse model of SYNGAP1-related disorders.

Molecular therapy : the journal of the American Society of Gene Therapy - 3 Dec 2025

Quinlan Meagan A, Guo Rong, Clark Andrew G, Luber Emily M, Christian Robert J, Martinez Refugio A, Groce Erin L, Liu Jiatai, Bishaw Yemeserach M, Bhowmik Ravi, Liang Elizabeth, Reding Melissa, Ronellenfitch Kara, Wright Vonn, Gudsnuk Kathryn M, Leedy Jennifer M, Mich John K, Gore Bryan B, Daigle Tanya L, Lopez Manuel E, Lein Ed S, Ichida Justin K, Levi Boaz P

Abstract excerpt

SYNGAP1-related disorders (SRDs) are rare neurodevelopmental conditions characterized by severe neurological symptoms, including epilepsy, motor impairment, and cognitive dysfunction. Current treatment options are limited, with patients relying on a cocktail of medications to manage the diverse symptoms but that do not address the underlying pathology. SRDs are primarily caused by haploinsufficiency of the...

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Identifiers

PMID: 40988338
DOI: 10.1016/j.ymthe.2025.09.040
PMCID: PMC12703155

Genes

SYNGAP1

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