Induction of long-term tolerance to a specific antigen using anti-CD3 lipid nanoparticles following gene therapy

por Chen, Chun-Yu, Vander Kooi, Amber, Cavedon, Alex, Cai, Xiaohe, Hoggatt, Jonathan, Martini, Paolo G.V., Miao, Carol H.
Publicado 2023

Treatment of Hemophilia A Using Factor VIII Messenger RNA Lipid Nanoparticles

por Chen, Chun-Yu, Tran, Dominic M., Cavedon, Alex, Cai, Xiaohe, Rajendran, Raj, Lyle, Meghan J., Martini, Paolo G.V., Miao, Carol H.
Publicado 2020

Lipid nanoparticle-encapsulated mRNA therapy corrects serum total bilirubin level in Crigler-Najjar syndrome mouse model

por Greig, Jenny A., Chorazeczewski, Joanna K., Chowdhary, Vivek, Smith, Melanie K., Jennis, Matthew, Tarrant, James C., Buza, Elizabeth L., Coughlan, Kimberly, Martini, Paolo G.V., Wilson, James M.
Publicado 2023

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

Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency

por Khoja, Suhail, Liu, Xiao-Bo, Truong, Brian, Nitzahn, Matthew, Lambert, Jenna, Eliav, Adam, Nasser, Eram, Randolph, Emma, Burke, Kristine E., White, Rebecca, Zhu, Xuling, Martini, Paolo G.V., Nissim, Itzhak, Cederbaum, Stephen D., Lipshutz, Gerald S.
Publicado 2022

Synthetic human ABCB4 mRNA therapy rescues severe liver disease phenotype in a BALB/c.Abcb4(−/−) mouse model of PFIC3

por Wei, Guangyan, Cao, Jingsong, Huang, Pinzhu, An, Ping, Badlani, Disha, Vaid, Kahini A., Zhao, Shuangshuang, Wang, David Q-H., Zhuo, Jenny, Yin, Ling, Frassetto, Andrea, Markel, Arianna, Presnyak, Vladimir, Gandham, Srujan, Hua, Serenus, Lukacs, Christine, Finn, Patrick F., Giangrande, Paloma H., Martini, Paolo G.V., Popov, Yury V.
Publicado 2020

mRNA-based therapy in a rabbit model of variegate porphyria offers new insights into the pathogenesis of acute attacks

por Jericó, Daniel, Córdoba, Karol M., Jiang, Lei, Schmitt, Caroline, Morán, María, Sampedro, Ana, Alegre, Manuel, Collantes, María, Santamaría, Eva, Alegre, Estíbaliz, Culerier, Corinne, de Mendoza, Ander Estella-Hermoso, Oyarzabal, Julen, Martín, Miguel A., Peñuelas, Iván, Ávila, Matías A., Gouya, Laurent, Martini, Paolo G.V., Fontanellas, Antonio
Publicado 2021

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

GLA-modified RNA treatment lowers GB3 levels in iPSC-derived cardiomyocytes from Fabry-affected individuals

por ter Huurne, Menno, Parker, Benjamin L., Liu, Ning Qing, Qian, Elizabeth Ling, Vivien, Celine, Karavendzas, Kathy, Mills, Richard J., Saville, Jennifer T., Abu-Bonsrah, Dad, Wise, Andrea F., Hudson, James E., Talbot, Andrew S., Finn, Patrick F., Martini, Paolo G.V., Fuller, Maria, Ricardo, Sharon D., Watt, Kevin I., Nicholls, Kathy M., Porrello, Enzo R., Elliott, David A.
Publicado 2023

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia

por An, Ding, Schneller, Jessica L., Frassetto, Andrea, Liang, Shi, Zhu, Xuling, Park, Ji-Sun, Theisen, Matt, Hong, Sue-Jean, Zhou, Jenny, Rajendran, Raj, Levy, Becca, Howell, Rebecca, Besin, Gilles, Presnyak, Vladimir, Sabnis, Staci, Murphy-Benenato, Kerry E., Kumarasinghe, E. Sathyajith, Salerno, Timothy, Mihai, Cosmin, Lukacs, Christine M., Chandler, Randy J., Guey, Lin T., Venditti, Charles P., Martini, Paolo G.V.
Publicado 2017