Age-sensitive response of systemic AAV-mediated gene therapy in a newly characterized feline model of mucolipidosis II.
在新鉴定的猫粘脂病 II 型模型中，系统性 AAV 介导的基因治疗的年龄敏感性反应
期刊:Molecular Therapy
影响因子:12
doi:10.1016/j.ymthe.2025.04.030
Pyne Nettie K, Bagel Jessica, Shyng Charles, Odonnell Patricia, Miyadera Keiko, Srnak Jennifer, Swain Gary, Pesayco Jill P, Shelton G Diane, Assenmacher Charles-Antoine, Dickson Patricia, Stern Joshua A, Flanagan-Steet Heather, Gray Steven J, Bradbury Allison M
