Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy

por DeRosa, Frank, Smith, Lianne, Shen, Yinghua, Huang, Yan, Pan, Jing, Xie, Hongsheng, Yahalom, Barak, Heartlein, Michael W.
Publicado 2019

CNS Penetration of Intrathecal-Lumbar Idursulfase in the Monkey, Dog and Mouse: Implications for Neurological Outcomes of Lysosomal Storage Disorder

por Calias, Pericles, Papisov, Mikhail, Pan, Jing, Savioli, Nancy, Belov, Vasily, Huang, Yan, Lotterhand, Jason, Alessandrini, Mary, Liu, Nan, Fischman, Alan J., Powell, Jan L., Heartlein, Michael W.
Publicado 2012

Human Sulfatase 2 inhibits in vivo tumor growth of MDA-MB-231 human breast cancer xenografts

por Peterson, Sarah M, Iskenderian, Andrea, Cook, Lynette, Romashko, Alla, Tobin, Kristen, Jones, Michael, Norton, Angela, Gómez-Yafal, Alicia, Heartlein, Michael W, Concino, Michael F, Liaw, Lucy, Martini, Paolo GV
Publicado 2010

Recombinant human complement component C2 produced in a human cell line restores the classical complement pathway activity in-vitro: an alternative treatment for C2 deficiency dise...

por Martini, Paolo GV, Cook, Lynette C, Alderucci, Scott, Norton, Angela W, Lundberg, Dianna M, Fish, Susan M, Langsetmo, Knut, Jönsson, Göran, Lood, Christian, Gullstrand, Birgitta, Zaleski, Kate J, Savioli, Nancy, Lottherand, Jason, Bedard, Charles, Gill, John, Concino, Michael F, Heartlein, Michael W, Truedsson, Lennart, Powell, Jan L, Tzianabos, Arthur O
Publicado 2010