Inhaled Nanoformulated mRNA Polyplexes for Protein Production in Lung Epithelium von Patel, Asha Kumari, Kaczmarek, James C., Bose, Suman, Kauffman, Kevin J., Mir, Faryal, Heartlein, Michael W., DeRosa, Frank, Langer, Robert, Anderson, Daniel G.

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Polymer-Lipid Nanoparticles for Systemic Delivery of mRNA to the Lungs von Kaczmarek, James C., Patel, Asha K., Kauffman, Kevin J., Fenton, Owen S., Webber, Matthew J., Heartlein, Michael W., DeRosa, Frank, Anderson, Daniel G.

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Optimization of a Degradable Polymer–Lipid Nanoparticle for Potent Systemic Delivery of mRNA to the Lung Endothelium and Immune Cells von Kaczmarek, James C, Kauffman, Kevin J, Fenton, Owen S, Sadtler, Kaitlyn, Patel, Asha K, Heartlein, Michael W, DeRosa, Frank, Anderson, Daniel G

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Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy von DeRosa, Frank, Smith, Lianne, Shen, Yinghua, Huang, Yan, Pan, Jing, Xie, Hongsheng, Yahalom, Barak, Heartlein, Michael W.

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Synthesis and Biological Evaluation of Ionizable Lipid Materials for the In Vivo Delivery of Messenger RNA to B Lymphocytes von Fenton, Owen S., Kauffman, Kevin J., Kaczmarek, James C., McClellan, Rebecca L., Jhunjhunwala, Siddharth, Tibbitt, Mark W., Zeng, Manhao D., Appel, Eric A., Dorkin, Joseph R., Mir, Faryal F., Yang, Jung H., Oberli, Matthias A., Heartlein, Michael W., DeRosa, Frank, Langer, Robert, Anderson, Daniel G.

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Customizable Lipid Nanoparticle Materials for the Delivery of siRNAs and mRNAs von Fenton, Owen S., Kauffman, Kevin J., McClellan, Rebecca L., Kaczmarek, James C., Zeng, Manhao D., Andresen, Jason L., Rhym, Luke H., Heartlein, Michael W., DeRosa, Frank, Anderson, Daniel G.

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CNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorder von 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

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mRNA Delivery for Therapeutic Anti-HER2 Antibody Expression In Vivo von Rybakova, Yulia, Kowalski, Piotr S., Huang, Yuxuan, Gonzalez, John T., Heartlein, Michael W., DeRosa, Frank, Delcassian, Derfogail, Anderson, Daniel G.

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Optimization of Lipid Nanoparticle Formulations for mRNA Delivery in Vivo with Fractional Factorial and Definitive Screening Designs von Kauffman, Kevin J, Dorkin, J. Robert, Yang, Jung H, Heartlein, Michael W, DeRosa, Frank, Mir, Faryal F, Fenton, Owen S, Anderson, Daniel G

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Systemic delivery of mRNA and DNA to the lung using polymer-lipid nanoparticles von Kaczmarek, James C., Patel, Asha Kumari, Rhym, Luke H., Palmiero, Umberto Capasso, Bhat, Balkrishen, Heartlein, Michael W., DeRosa, Frank, Anderson, Daniel G.

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Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery von Fenton, Owen S., Kauffman, Kevin J., McClellan, Rebecca L., Appel, Eric A., Dorkin, J. Robert, Tibbitt, Mark W., Heartlein, Michael W., DeRosa, Frank, Langer, Robert, Anderson, Daniel G.

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Efficacy and immunogenicity of unmodified and pseudouridine-modified mRNA delivered systemically with lipid nanoparticles in vivo von Kauffman, Kevin J, Mir, Faryal F, Jhunjhunwala, Siddharth, Kaczmarek, James C, Hurtado, Juan E, Yang, Jung H, Webber, Matthew J, Kowalski, Piotr S, Heartlein, Michael W, DeRosa, Frank, Anderson, Daniel G

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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

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Gene Delivery: Inhaled Nanoformulated mRNA Polyplexes for Protein Production in Lung Epithelium (Adv. Mater. 8/2019) von Patel, Asha Kumari, Kaczmarek, James C., Bose, Suman, Kauffman, Kevin J., Mir, Faryal, Heartlein, Michael W., DeRosa, Frank, Langer, Robert, Anderson, Daniel G.

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Erythropoietin Produced in a Human Cell Line (Dynepo) Has Significant Differences in Glycosylation Compared with Erythropoietins Produced in CHO Cell Lines von Shahrokh, Zahra, Royle, Louise, Saldova, Radka, Bones, Jonathan, Abrahams, Jodie L, Artemenko, Natalia V, Flatman, Steve, Davies, Mike, Baycroft, Alison, Sehgal, Surinder, Heartlein, Michael W, Harvey, David J, Rudd, Pauline M

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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... von Martini, Paolo G V, 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

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Safety Evaluation of Chronic Intrathecal Administration of Idursulfase-IT in Cynomolgus Monkeys von Felice, Brian R., Wright, Teresa L., Boyd, Robert B., Butt, Mark T., Pfeifer, Richard W., Pan, Jing, Ruiz, Juan A., Heartlein, Michael W., Calias, Pericles

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Customizable Lipid Nanoparticle Materials for the Delivery of siRNAs and mRNAs von Fenton, Owen S., Kauffman, Kevin J., McClellan, Rebecca L., Kaczmarek, James C., Zeng, Manhao D., Andresen, Jason L., Rhym, Luke H., Heartlein, Michael W., DeRosa, Frank, Anderson, Daniel G.

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Polymer-Lipid Nanoparticles for Systemic Delivery of mRNA to the Lungs von Kaczmarek, James C., Patel, Asha K., Kauffman, Kevin J., Fenton, Owen S., Webber, Matthew J., Heartlein, Michael W., DeRosa, Frank, Anderson, Daniel G.

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Safety evaluation of chronic intrathecal administration of heparan N-sulfatase in juvenile cynomolgus monkeys von Pfeifer, Richard W., Felice, Brian R., Boyd, Robert B., Butt, Mark T., Ruiz, Juan A., Heartlein, Michael W., Calias, Pericles

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