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10 results on '"Kauffman, Kevin"'

1. NAD depletion mediates cytotoxicity in human neurons with autophagy deficiency

Author

Sun, Congxin, primary, Seranova, Elena, additional, Cohen, Malkiel A., additional, Chipara, Miruna, additional, Roberts, Jennie, additional, Astuti, Dewi, additional, Palhegyi, Adina M., additional, Acharjee, Animesh, additional, Sedlackova, Lucia, additional, Kataura, Tetsushi, additional, Otten, Elsje G., additional, Panda, Prashanta K., additional, Lara-Reyna, Samuel, additional, Korsgen, Miriam E., additional, Kauffman, Kevin J., additional, Huerta-Uribe, Alejandro, additional, Zatyka, Malgorzata, additional, Silva, Luiz F.S.E., additional, Torresi, Jorge, additional, Zhang, Shupei, additional, Hughes, Georgina W., additional, Ward, Carl, additional, Kuechler, Erich R., additional, Cartwright, David, additional, Trushin, Sergey, additional, Trushina, Eugenia, additional, Sahay, Gaurav, additional, Buganim, Yosef, additional, Lavery, Gareth G., additional, Gsponer, Joerg, additional, Anderson, Daniel G., additional, Frickel, Eva-Maria, additional, Rosenstock, Tatiana R., additional, Barrett, Timothy, additional, Maddocks, Oliver D.K., additional, Tennant, Daniel A., additional, Wang, Haoyi, additional, Jaenisch, Rudolf, additional, Korolchuk, Viktor I., additional, and Sarkar, Sovan, additional

Published
2023
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2. NAD Depletion Mediates Cytotoxicity in Human Neurons With Autophagy Deficiency

Author

Sun, Congxin, primary, Seranova, Elena, additional, Cohen, Malkiel A., additional, Chipara, Miruna, additional, Roberts, Jennie, additional, Palhegyi, Adina M., additional, Acharjee, Animesh, additional, Sedlackova, Lucia, additional, Kataura, Tetsushi, additional, Otten, Elsje G., additional, Panda, Prashanta K., additional, Kauffman, Kevin J., additional, Huerta-Uribe, Alejandro, additional, Zatyka, Malgorzata, additional, Silva, Luiz F.S.E., additional, Torresi, Jorge, additional, Zhang, Shupei, additional, Ward, Carl, additional, Kuechler, Erich R., additional, Cartwright, David, additional, Trushin, Sergey, additional, Trushina, Eugenia, additional, Sahay, Gaurav, additional, Buganim, Yosef, additional, Lavery, Gareth G., additional, Gsponer, Joerg, additional, Anderson, Daniel G., additional, Rosenstock, Tatiana R., additional, Barrett, Timothy, additional, Maddocks, Oliver D.K., additional, Tennant, Daniel A., additional, Wang, Haoyi, additional, Jaenisch, Rudolf, additional, Korolchuk, Viktor I., additional, and Sarkar, Sovan, additional

Published
2022
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3. In Vivo RNAi-Mediated eIF3m Knockdown Affects Ribosome Biogenesis and Transcription but Has Limited Impact on mRNA-Specific Translation

Author

Smekalova, Elena M., primary, Gerashchenko, Maxim V., additional, O’Connor, Patrick B.F., additional, Whittaker, Charles A., additional, Kauffman, Kevin J., additional, Fefilova, Anna S., additional, Zatsepin, Timofei S., additional, Bogorad, Roman L., additional, Baranov, Pavel V., additional, Langer, Robert, additional, Gladyshev, Vadim N., additional, Anderson, Daniel G., additional, and Koteliansky, Victor, additional

Published
2020
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5. Identification of Novel Fibrosis Modifiers by In Vivo siRNA Silencing

Author

Vollmann, Elisabeth H., primary, Cao, Lizhi, additional, Amatucci, Aldo, additional, Reynolds, Taylor, additional, Hamann, Stefan, additional, Dalkilic-Liddle, Isin, additional, Cameron, Thomas O., additional, Hossbach, Markus, additional, Kauffman, Kevin J., additional, Mir, Faryal F., additional, Anderson, Daniel G., additional, Novobrantseva, Tatiana, additional, Koteliansky, Victor, additional, Kisseleva, Tatiana, additional, Brenner, David, additional, Duffield, Jeremy, additional, and Burkly, Linda C., additional

Published
2017
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6. Cell-Cycle-Targeting MicroRNAs as Therapeutic Tools against Refractory Cancers

Author

Hydbring, Per, primary, Wang, Yinan, additional, Fassl, Anne, additional, Li, Xiaoting, additional, Matia, Veronica, additional, Otto, Tobias, additional, Choi, Yoon Jong, additional, Sweeney, Katharine E., additional, Suski, Jan M., additional, Yin, Hao, additional, Bogorad, Roman L., additional, Goel, Shom, additional, Yuzugullu, Haluk, additional, Kauffman, Kevin J., additional, Yang, Junghoon, additional, Jin, Chong, additional, Li, Yingxiang, additional, Floris, Davide, additional, Swanson, Richard, additional, Ng, Kimmie, additional, Sicinska, Ewa, additional, Anders, Lars, additional, Zhao, Jean J., additional, Polyak, Kornelia, additional, Anderson, Daniel G., additional, Li, Cheng, additional, and Sicinski, Piotr, additional

Published
2017
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7. Efficacy and immunogenicity of unmodified and pseudouridine-modified mRNA delivered systemically with lipid nanoparticles in vivo

Author

Kauffman, Kevin J., primary, Mir, Faryal F., additional, Jhunjhunwala, Siddharth, additional, Kaczmarek, James C., additional, Hurtado, Juan E., additional, Yang, Jung H., additional, Webber, Matthew J., additional, Kowalski, Piotr S., additional, Heartlein, Michael W., additional, DeRosa, Frank, additional, and Anderson, Daniel G., additional

Published
2016
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10. Efficacy and immunogenicity of unmodified and pseudouridine-modified mRNA delivered systemically with lipid nanoparticles in vivo

Author

Kevin J. Kauffman, Michael W. Heartlein, Daniel G. Anderson, Matthew J. Webber, Piotr S. Kowalski, Juan E. Hurtado, Frank Derosa, Siddharth Jhunjhunwala, James C. Kaczmarek, Faryal F. Mir, Jung H. Yang, Institute for Medical Engineering and Science, Harvard University--MIT Division of Health Sciences and Technology, Massachusetts Institute of Technology. Department of Chemical Engineering, Koch Institute for Integrative Cancer Research at MIT, Kauffman, Kevin John, Mir, Faryal, Jhunjhunwala, Siddharth, Kaczmarek, James Cliff, Hurtado, Juan E., Yang, Jung H, Webber, Matthew, Kowalski, Piotr S, and Anderson, Daniel Griffith

Subjects
0301 basic medicine, Surface Properties, Biophysics, Gene Expression, Bioengineering, Pharmacology, Biology, Transfection, Pseudouridine, Article, Biomaterials, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Immune system, In vivo, Extracellular, Animals, Humans, Myeloid Cells, RNA, Messenger, Particle Size, Messenger RNA, Innate immune system, Immunogenicity, Gene Transfer Techniques, Molecular biology, Lipids, Uridine, Immunity, Innate, Mice, Inbred C57BL, 030104 developmental biology, chemistry, Liver, Mechanics of Materials, 030220 oncology & carcinogenesis, Ceramics and Composites, Cytokines, Nanoparticles, Female, HeLa Cells
Abstract

mRNA has broad potential for treating diseases requiring protein expression. However, mRNA can also induce an immune response with associated toxicity. Replacement of uridine bases with pseudouridine has been postulated to modulate both mRNA immunogenicity and potency. Here, we explore the immune response and activity of lipid nanoparticle-formulated unmodified and pseudouridine-modified mRNAs administered systemically in vivo. Pseudouridine modification to mRNA had no significant effect on lipid nanoparticle physical properties, protein expression in vivo, or mRNA immunogenicity compared to unmodified mRNA when delivered systemically with liver-targeting lipid nanoparticles, but reduced in vitro transfection levels. Indicators of a transient, extracellular innate immune response to mRNA were observed, including neutrophilia, myeloid cell activation, and up-regulation of four serum cytokines. This study provides insight into the immune responses to mRNA lipid nanoparticles, and suggests that pseudouridine modifications may be unnecessary for therapeutic application of mRNA in the liver. Keywords: mRNA, Base modification, Pseudouridine, Lipid nanoparticle, In vivo, Immmune response, Shire Pharmaceuticals

Published
2016

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