Litcius/Paper detail

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

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

2025Molecular Therapy10 citationsDOIOpen Access PDF

Topics & Concepts

HaploinsufficiencyPhenotypeMedicineNeurodevelopmental disorderNeuroscienceImmunologyDiseaseGenetic enhancementBioinformaticsEpilepsyDownregulation and upregulationCognitionClinical trialGene deliveryGeneBiologySynapsinViral vectorGenetics and Neurodevelopmental DisordersLysosomal Storage Disorders ResearchCarbohydrate Chemistry and Synthesis