Neuron-targeted gene therapy rescues multiple phenotypes of STXBP1-related disorders in mice and is well tolerated in nonhuman primates.
针对神经元的基因疗法可以挽救小鼠中多种 STXBP1 相关疾病的表型，并且在非人灵长类动物中具有良好的耐受性
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.05.011
Aeran Rangoli, Tanenhaus Annie, Sears Sheila M S, Moerke Nathan J, Miller Adam, Artur Camille, Bouhlal Yosr, Bove Peter F, Diaz de Arce Alexander J, Shimizu Saki, Le Jason, Place Keith, Hoffelt Dixon, Amarlkhagva Tselmeg, Su Jennifer, Chen Ming, Babineau Brooke A, McLaughlin John, Soe Myat, Macdonald Warren, Lin I Winnie, Bole Dhruv, Valentine Kristen M, Hallam Elizabeth, Dhanota Puja, Liu Serena, Tan Steven A, Zhao Ben, Hosur Raghavendra, Vila Maria Candida, Poda Suresh, Belle Archana, Tagliatela Stephanie
