Characterization of Fluid Biomarkers Reveals Lysosome Dysfunction and Neurodegeneration in Neuronopathic MPS II Patients

por Bhalla, Akhil, Ravi, Ritesh, Fang, Meng, Arguello, Annie, Davis, Sonnet S., Chiu, Chi-Lu, Blumenfeld, Jessica R., Nguyen, Hoang N., Earr, Timothy K., Wang, Junhua, Astarita, Giuseppe, Zhu, Yuda, Fiore, Damian, Scearce-Levie, Kimberly, Diaz, Dolores, Cahan, Heather, Troyer, Matthew D., Harris, Jeffrey M., Escolar, Maria L.
Publicado 2020

Iduronate-2-sulfatase transport vehicle rescues behavioral and skeletal phenotypes in a mouse model of Hunter syndrome

por Arguello, Annie, Meisner, René, Thomsen, Elliot R., Nguyen, Hoang N., Ravi, Ritesh, Simms, Jeffrey, Lo, Iris, Speckart, Jessica, Holtzman, Julia, Gill, Thomas M., Chan, Darren, Cheng, Yuhsiang, Chiu, Chi-Lu, Dugas, Jason C., Fang, Meng, Lopez, Isabel A., Solanoy, Hilda, Tsogtbaatar, Buyankhishig, Zhu, Yuda, Bhalla, Akhil, Henne, Kirk R., Henry, Anastasia G., Delucchi, Anthony, Costanzo, Simona, Harris, Jeffrey M., Diaz, Dolores, Scearce-Levie, Kimberly, Sanchez, Pascal E.
Publicado 2021