BB-301: a silence and replace AAV-based vector for the treatment of oculopharyngeal muscular dystrophy
BB-301:一种用于治疗眼咽型肌营养不良症的沉默和替代 AAV 载体
期刊:Molecular Therapy-Nucleic Acids
影响因子:6.5
doi:10.1016/j.omtn.2021.02.017
Vanessa Strings-Ufombah, Alberto Malerba, Shih-Chu Kao, Sonal Harbaran, Fanny Roth, Ornella Cappellari, Ngoc Lu-Nguyen, Keiko Takahashi, Sophie Mukadam, Georgina Kilfoil, Claudia Kloth, Petrus Roelvink, George Dickson, Capucine Trollet, David Suhy