A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis
Titel:
A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis
Auteur:
Kell, Pamela Sidhu, Rohini Qian, Mingxing Mishra, Sonali Nicoli, Elena-Raluca D'Souza, Precilla Tifft, Cynthia J. Gross, Amanda L. Gray-Edwards, Heather L. Martin, Douglas R. Sena- Esteves, Miguel Dietzen, Dennis J. Singh, Manmilan Luo, Jingqin Schaffer, Jean E. Ory, Daniel S. Jiang, Xuntian