Treatment of skeletal and non-skeletal alterations of Mucopolysaccharidosis type IVA by AAV-mediated gene therapy
利用腺相关病毒介导的基因疗法治疗IVA型粘多糖贮积症的骨骼和非骨骼病变
期刊:Nature Communications
影响因子:15.7
doi:10.1038/s41467-021-25697-y
Bertolin, Joan; Sánchez, Víctor; Ribera, Albert; Jaén, Maria Luisa; Garcia, Miquel; Pujol, Anna; Sánchez, Xavier; Muñoz, Sergio; Marcó, Sara; Pérez, Jennifer; Elias, Gemma; León, Xavier; Roca, Carles; Jimenez, Veronica; Otaegui, Pedro; Mulero, Francisca; Navarro, Marc; Ruberte, Jesús; Bosch, Fatima