Search Results - "Heartlein, Michael"

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

“…Noninvasive aerosol inhalation is an established method of drug delivery to the lung, and remains a desirable route for nucleic‐acid‐based therapeutics. In…”
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Polymer-Lipid Nanoparticles for Systemic Delivery of mRNA to the Lungs by Kaczmarek, James C., Patel, Asha K., Kauffman, Kevin J., Fenton, Owen S., Webber, Matthew J., Heartlein, Michael W., DeRosa, Frank, Anderson, Daniel G.

“…Therapeutic nucleic acids hold great promise for the treatment of disease but require vectors for safe and effective delivery. Synthetic nanoparticle vectors…”
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Optimization of a Degradable Polymer–Lipid Nanoparticle for Potent Systemic Delivery of mRNA to the Lung Endothelium and Immune Cells by Kaczmarek, James C, Kauffman, Kevin J, Fenton, Owen S, Sadtler, Kaitlyn, Patel, Asha K, Heartlein, Michael W, DeRosa, Frank, Anderson, Daniel G

“…mRNA therapeutics hold great potential for treating a variety of diseases through protein-replacement, immunomodulation, and gene editing. However, much like…”
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mRNA Delivery for Therapeutic Anti-HER2 Antibody Expression In Vivo by Rybakova, Yulia, Kowalski, Piotr S., Huang, Yuxuan, Gonzalez, John T., Heartlein, Michael W., DeRosa, Frank, Delcassian, Derfogail, Anderson, Daniel G.

“…Antibody-based drugs are a leading class of biologics used to treat a variety of diseases, including cancer. However, wide antibody implementation is hindered…”
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Improved Efficacy in a Fabry Disease Model Using a Systemic mRNA Liver Depot System as Compared to Enzyme Replacement Therapy by DeRosa, Frank, Smith, Lianne, Shen, Yinghua, Huang, Yan, Pan, Jing, Xie, Hongsheng, Yahalom, Barak, Heartlein, Michael W.

“…Fabry disease is a lysosomal storage disorder caused by the deficiency of α-galactosidase A. Enzyme deficiency results in a progressive decline in renal and…”
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Optimization of Lipid Nanoparticle Formulations for mRNA Delivery in Vivo with Fractional Factorial and Definitive Screening Designs by Kauffman, Kevin J, Dorkin, J. Robert, Yang, Jung H, Heartlein, Michael W, DeRosa, Frank, Mir, Faryal F, Fenton, Owen S, Anderson, Daniel G

“…Intracellular delivery of messenger RNA (mRNA) has the potential to induce protein production for many therapeutic applications. Although lipid nanoparticles…”
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Systemic delivery of mRNA and DNA to the lung using polymer-lipid nanoparticles by Kaczmarek, James C., Patel, Asha Kumari, Rhym, Luke H., Palmiero, Umberto Capasso, Bhat, Balkrishen, Heartlein, Michael W., DeRosa, Frank, Anderson, Daniel G.

“…Non-viral vectors offer the potential to deliver nucleic acids including mRNA and DNA into cells in vivo. However, designing materials that effectively deliver…”
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Synthesis and Biological Evaluation of Ionizable Lipid Materials for the In Vivo Delivery of Messenger RNA to B Lymphocytes by 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.

“…B lymphocytes regulate several aspects of immunity including antibody production, cytokine secretion, and T‐cell activation; moreover, B cell misregulation is…”
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CNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorder by 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

“…A major challenge for the treatment of many central nervous system (CNS) disorders is the lack of convenient and effective methods for delivering biological…”
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Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery by 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.

“…Thousands of human diseases could be treated by selectively controlling the expression of specific proteins in vivo. A new series of alkenyl amino alcohol…”
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Customizable Lipid Nanoparticle Materials for the Delivery of siRNAs and mRNAs by 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.

“…RNAs are a promising class of therapeutics given their ability to regulate protein concentrations at the cellular level. Developing safe and effective…”
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Efficacy and immunogenicity of unmodified and pseudouridine-modified mRNA delivered systemically with lipid nanoparticles in vivo by 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

“…Abstract mRNA has broad potential for treating diseases requiring protein expression. However, mRNA can also induce an immune response with associated…”
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Poly(glycoamidoamine) Brushes Formulated Nanomaterials for Systemic siRNA and mRNA Delivery in Vivo by Dong, Yizhou, Dorkin, J. Robert, Wang, Weiheng, Chang, Philip H, Webber, Matthew J, Tang, Benjamin C, Yang, Junghoon, Abutbul-Ionita, Inbal, Danino, Dganit, DeRosa, Frank, Heartlein, Michael, Langer, Robert, Anderson, Daniel G

“…Safe and effective delivery is required for siRNA and mRNA-based therapeutics to reach their potential. Here, we report on the development of…”
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Gene Delivery: Inhaled Nanoformulated mRNA Polyplexes for Protein Production in Lung Epithelium (Adv. Mater. 8/2019) by Patel, Asha Kumari, Kaczmarek, James C., Bose, Suman, Kauffman, Kevin J., Mir, Faryal, Heartlein, Michael W., DeRosa, Frank, Langer, Robert, Anderson, Daniel G.

“…In vitro transcribed messenger RNA is nebulized using poly(beta amino esters) for inhalable delivery to the lung as described by Daniel G. Anderson and…”
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Human Sulfatase 2 inhibits in vivo tumor growth of MDA-MB-231 human breast cancer xenografts by 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

“…Extracellular human sulfatases modulate growth factor signaling by alteration of the heparin/heparan sulfate proteoglycan (HSPG) 6-O-sulfation state. HSPGs…”
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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 by 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

“…Recombinant human erythropoietin has been used to treat anemia associated with chronic renal disease. This paper provides a comprehensive comparative analysis…”
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Safety Evaluation of Chronic Intrathecal Administration of Idursulfase-IT in Cynomolgus Monkeys by 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

“…Recombinant human idursulfase, an intravenous enzyme replacement therapy indicated for treatment of somatic symptoms of mucopolysaccharidosis II (Hunter…”
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Customizable Lipid Nanoparticle Materials for the Delivery of siRNAs and mRNAs by 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.

“…RNAs are a promising class of therapeutics given their ability to regulate protein concentrations at the cellular level. Developing safe and effective…”
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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 diseases by 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

“…Complement C2 deficiency is the most common genetically determined complete complement deficiency and is associated with a number of diseases. Most prominent…”
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Polymer-Lipid Nanoparticles for Systemic Delivery of mRNA to the Lungs by Kaczmarek, James C., Patel, Asha K., Kauffman, Kevin J., Fenton, Owen S., Webber, Matthew J., Heartlein, Michael W., DeRosa, Frank, Anderson, Daniel G.

“…Therapeutic nucleic acids hold great promise for the treatment of disease but require vectors for safe and effective delivery. Synthetic nanoparticle vectors…”
Get full text