AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study
AAV介导的基因疗法治疗半乳糖唾液酸缺乏症:一项长期安全性和有效性研究
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2021.10.007
Hu, Huimin; Mosca, Rosario; Gomero, Elida; van de Vlekkert, Diantha; Campos, Yvan; Fremuth, Leigh E; Brown, Scott A; Weesner, Jason A; Annunziata, Ida; d'Azzo, Alessandra