Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe's Disease after Global Gene Therapy
克拉伯氏病小鼠模型接受整体基因治疗后神经系统症状和代谢功能障碍得到长期改善
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2018.01.009
Michael S Marshall, Yazan Issa, Benas Jakubauskas, Monika Stoskute, Vince Elackattu, Jeffrey N Marshall, Wil Bogue, Duc Nguyen, Zane Hauck, Emily Rue, Subha Karumuthil-Melethil, Violeta Zaric, Maarten Bosland, Richard B van Breemen, Maria I Givogri, Steven J Gray, Stephen J Crocker, Ernesto R Bongar