AAV delivery of full-length SYNGAP1 rescues epileptic and behavioral phenotypes in a mouse model of SYNGAP1-related disorders
在SYNGAP1相关疾病的小鼠模型中,通过腺相关病毒(AAV)递送全长SYNGAP1可挽救癫痫和行为表型。
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2025.09.040
Meagan A Quinlan,Rong Guo,Andrew G Clark,Emily M Luber,Robert J Christian,Refugio A Martinez,Erin L Groce,Jiatai Liu,Yemeserach M Bishaw,Ravi Bhowmik,Elizabeth Liang,Melissa Reding,Kara Ronellenfitch,Vonn Wright,Kathryn M 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