A pentasaccharide for monitoring pharmacodynamic response to gene therapy in GM1 gangliosidosis

Pamela Kell, Rohini Sidhu, Mingxing Qian, Sonali Mishra, Elena Raluca Nicoli, Precilla D'Souza, Cynthia J. Tifft, Amanda L. Gross, Heather L. Gray-Edwards, Douglas R. Martin, Miguel Sena-Esteves, Dennis J. Dietzen, Manmilan Singh, Jingqin Luo, Jean E. Schaffer, Daniel Ory, Xuntian Jiang

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