Characterizing the mechanism of action for mRNA therapeutics for the treatment of propionic acidemia, methylmalonic acidemia, and phenylketonuria von Baek, Rena, Coughlan, Kimberly, Jiang, Lei, Liang, Min, Ci, Lei, Singh, Harkewal, Zhang, Hannah, Kaushal, Neeraj, Rajlic, Ivana Liric, Van, Linh, Dimen, Rain, Cavedon, Alexander, Yin, Ling, Rice, Lisa, Frassetto, Andrea, Guey, Lin, Finn, Patrick, Martini, Paolo G. V.

Volltext

Messenger RNA therapy for rare genetic metabolic diseases von Berraondo, Pedro, Martini, Paolo G V, Avila, Matias A, Fontanellas, Antonio

Volltext

Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency von Truong, Brian, Allegri, Gabriella, Liu, Xiao-Bo, Burke, Kristine E., Zhu, Xuling, Cederbaum, Stephen D., Häberle, Johannes, Martini, Paolo G. V., Lipshutz, Gerald S.

Volltext

Dual mRNA therapy restores metabolic function in long-term studies in mice with propionic acidemia von Jiang, Lei, Park, Ji-Sun, Yin, Ling, Laureano, Rodrigo, Jacquinet, Eric, Yang, Jinsong, Liang, Shi, Frassetto, Andrea, Zhuo, Jenny, Yan, Xinhua, Zhu, Xuling, Fortucci, Steven, Hoar, Kara, Mihai, Cosmin, Tunkey, Christopher, Presnyak, Vlad, Benenato, Kerry E., Lukacs, Christine M., Martini, Paolo G. V., Guey, Lin T.

Volltext

Recent Insights into the Pathogenesis of Acute Porphyria Attacks and Increasing Hepatic PBGD as an Etiological Treatment von Jericó, Daniel, Córdoba, Karol M, Sampedro, Ana, Jiang, Lei, Joucla, Gilles, Cabanne, Charlotte, Lanciego, José Luis, Martini, Paolo G V, Berraondo, Pedro, Ávila, Matías A, Fontanellas, Antonio

Volltext

Novel mRNA-Based Therapy Reduces Toxic Galactose Metabolites and Overcomes Galactose Sensitivity in a Mouse Model of Classic Galactosemia von Balakrishnan, Bijina, An, Ding, Nguyen, Vi, DeAntonis, Christine, Martini, Paolo G.V., Lai, Kent

Volltext

mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease von Cao, Jingsong, Choi, Minjung, Guadagnin, Eleonora, Soty, Maud, Silva, Marine, Verzieux, Vincent, Weisser, Edward, Markel, Arianna, Zhuo, Jenny, Liang, Shi, Yin, Ling, Frassetto, Andrea, Graham, Anne-Renee, Burke, Kristine, Ketova, Tatiana, Mihai, Cosmin, Zalinger, Zach, Levy, Becca, Besin, Gilles, Wolfrom, Meredith, Tran, Barbara, Tunkey, Christopher, Owen, Erik, Sarkis, Joe, Dousis, Athanasios, Presnyak, Vladimir, Pepin, Christopher, Zheng, Wei, Ci, Lei, Hard, Marjie, Miracco, Edward, Rice, Lisa, Nguyen, Vi, Zimmer, Mike, Rajarajacholan, Uma, Finn, Patrick F., Mithieux, Gilles, Rajas, Fabienne, Martini, Paolo G. V., Giangrande, Paloma H.

Volltext

Gonadotropin-releasing hormone agonists sensitize, and resensitize, prostate cancer cells to docetaxel in a p53-dependent manner von Moretti, Roberta M, Montagnani Marelli, Marina, Taylor, Deanne M, Martini, Paolo G V, Marzagalli, Monica, Limonta, Patrizia

Volltext

mRNA Therapy Improves Metabolic and Behavioral Abnormalities in a Murine Model of Citrin Deficiency von Cao, Jingsong, An, Ding, Galduroz, Mikel, Zhuo, Jenny, Liang, Shi, Eybye, Marianne, Frassetto, Andrea, Kuroda, Eishi, Funahashi, Aki, Santana, Jordan, Mihai, Cosmin, Benenato, Kerry E., Kumarasinghe, E. Sathyajith, Sabnis, Staci, Salerno, Timothy, Coughlan, Kimberly, Miracco, Edward J., Levy, Becca, Besin, Gilles, Schultz, Joshua, Lukacs, Christine, Guey, Lin, Finn, Patrick, Furukawa, Tatsuhiko, Giangrande, Paloma H., Saheki, Takeyori, Martini, Paolo G.V.

Volltext

Amnio acid substitution at position 298 of human glucose-6 phosphatase-α significantly impacts its stability in mammalian cells von Cao, Jingsong, Markel, Arianna, Hanahoe, Erin, Ketova, Tatiana, Mihai, Cosmin, Zalinger, Zach, Marquardt, David, Amato, Nicholas J., Cheng, Yi Min, Reid, David W., Dousis, Athanasios, Giangrande, Paloma H., Schultz, Joshua R., Martini, Paolo G. V., Finn, Patrick F.

Volltext

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

Volltext

Synthetic mRNA rescues very long-chain acyl-CoA dehydrogenase deficiency in patient fibroblasts and a murine model von Zhao, Xue-Jun, Mohsen, AI-Walid, Mihalik, Stephanie, Solo, Keaton, Aliu, Ermal, Shi, Huifang, Basu, Shakuntala, Kochersperger, Catherine, Van’t Land, Clinton, Karunanidhi, Anuradha, Coughlan, Kimberly A., Siddiqui, Summar, Rice, Lisa M., Hillier, Shawn, Guadagnin, Eleonora, Giangrande, Paloma H., Martini, Paolo G.V., Vockley, Jerry

Volltext

Estrogen Receptors α and β Mediate Distinct Pathways of Vascular Gene Expression, Including Genes Involved in Mitochondrial Electron Transport and Generation of Reactive Oxygen Spe... von O’Lone, Raegan, Knorr, Katrin, Jaffe, Iris Z, Schaffer, Michael E, Martini, Paolo G. V, Karas, Richard H, Bienkowska, Jadwiga, Mendelsohn, Michael E, Hansen, Ulla

Volltext

Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates von Zhu, Xuling, Yin, Ling, Theisen, Matt, Zhuo, Jenny, Siddiqui, Summar, Levy, Becca, Presnyak, Vladimir, Frassetto, Andrea, Milton, Jaclyn, Salerno, Timothy, Benenato, Kerry E., Milano, Joe, Lynn, Andy, Sabnis, Staci, Burke, Kristine, Besin, Gilles, Lukacs, Christine M., Guey, Lin T., Finn, Patrick F., Martini, Paolo G.V.

Volltext

Hepatocyte Nuclear Factor 4 alpha 2 Messenger RNA Reprograms Liver‐Enriched Transcription Factors and Functional Proteins in End‐Stage Cirrhotic Human Hepatocytes von Tafaleng, Edgar N., Mukherjee, Amitava, Bell, Aaron, Morita, Kazutoyo, Guzman‐Lepe, Jorge, Haep, Nils, Florentino, Rodrigo M., Diaz‐Aragon, Ricardo, Frau, Carla, Ostrowska, Alina, Schultz, Joshua R., Martini, Paolo G. V., Soto‐Gutierrez, Alejandro, Fox, Ira J.

Volltext

A New Era for Rare Genetic Diseases: Messenger RNA Therapy von Martini, Paolo G V, Guey, Lin T

Volltext

Systemic messenger RNA as an etiological treatment for acute intermittent porphyria von Jiang, Lei, Berraondo, Pedro, Jericó, Daniel, Guey, Lin T., Sampedro, Ana, Frassetto, Andrea, Benenato, Kerry E., Burke, Kristine, Santamaría, Eva, Alegre, Manuel, Pejenaute, Álvaro, Kalariya, Mayur, Butcher, William, Park, Ji-Sun, Zhu, Xuling, Sabnis, Staci, Kumarasinghe, E. Sathyajith, Salerno, Timothy, Kenney, Matthew, Lukacs, Christine M., Ávila, Matías A., Martini, Paolo G. V., Fontanellas, Antonio

Volltext

Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia von 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.

Volltext

Lipid Nanoparticle Packaging Is an Effective and Nontoxic mRNA Delivery Platform in Embryonic Zebrafish von Patton, Clay, Farr, Gist H., An, Ding, Martini, Paolo G.V., Maves, Lisa

Volltext

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

Volltext