日期:
2020 年 — 2026 年
2020
2021
2022
2023
2024
2025
2026
影响因子:

Optimising the IgG-degrading enzyme treatment regimen for enhanced adeno-associated virus transduction in the presence of neutralising antibodies

优化 IgG 降解酶治疗方案以在存在中和抗体的情况下增强腺相关病毒的转导

Irene Ros-Gañán, Mirja Hommel, Laia Trigueros-Motos, Blanche Tamarit, Estefanía Rodríguez-García, David Salas, Guiomar Pérez, Anne Douar, Jean Philippe Combal, Bernard Benichou, Veronica Ferrer, Gloria González-Aseguinolaza

Use of an adeno-associated virus serotype Anc80 to provide durable cure of phenylketonuria in a mouse model

使用腺相关病毒血清型 Anc80 在小鼠模型中提供苯丙酮尿症的持久治愈

Robert A Kaiser, Nicholas D Weber, Laia Trigueros-Motos, Kari L Allen, Michael Martinez, William Cao, Caitlin J VanLith, Lori G Hillin, Anne Douar, Gloria González-Aseguinolaza, Rafael Aldabe, Joseph B Lillegard

Gene therapy for progressive familial intrahepatic cholestasis type 3 in a clinically relevant mouse model

临床相关小鼠模型中对进行性家族性肝内胆汁淤积症 3 型进行基因治疗

Nicholas D Weber, Leticia Odriozola, Javier Martínez-García, Veronica Ferrer, Anne Douar, Bernard Bénichou, Gloria González-Aseguinolaza, Cristian Smerdou