Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy
在杜氏肌营养不良症GRMD模型中评估全身性AAV-微型肌营养不良蛋白基因治疗
期刊:Science Translational Medicine
影响因子:14.6
doi:10.1126/scitranslmed.abo1815
Birch, Sharla M; Lawlor, Michael W; Conlon, Thomas J; Guo, Lee-Jae; Crudele, Julie M; Hawkins, Eleanor C; Nghiem, Peter P; Ahn, Mihye; Meng, Hui; Beatka, Margaret J; Fickau, Brittany A; Prieto, Juan C; Styner, Martin A; Struharik, Michael J; Shanks, Courtney; Brown, Kristy J; Golebiowski, Diane; Bettis, Amanda K; Balog-Alvarez, Cynthia J; Clement, Nathalie; Coleman, Kirsten E; Corti, Manuela; Pan, Xiufang; Hauschka, Stephen D; Gonzalez, J Patrick; Morris, Carl A; Schneider, Joel S; Duan, Dongsheng; Chamberlain, Jeffrey S; Byrne, Barry J; Kornegay, Joe N