Search Results - "Hope, Michael J"

Sequence-engineered mRNA Without Chemical Nucleoside Modifications Enables an Effective Protein Therapy in Large Animals by Thess, Andreas, Grund, Stefanie, Mui, Barbara L, Hope, Michael J, Baumhof, Patrick, Fotin-Mleczek, Mariola, Schlake, Thomas

“…Being a transient carrier of genetic information, mRNA could be a versatile, flexible, and safe means for protein therapies. While recent findings highlight…”
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The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs by Akinc, Akin, Maier, Martin A., Manoharan, Muthiah, Fitzgerald, Kevin, Jayaraman, Muthusamy, Barros, Scott, Ansell, Steven, Du, Xinyao, Hope, Michael J., Madden, Thomas D., Mui, Barbara L., Semple, Sean C., Tam, Ying K., Ciufolini, Marco, Witzigmann, Dominik, Kulkarni, Jayesh A., van der Meel, Roy, Cullis, Pieter R.

“…The regulatory approval of Onpattro, a lipid nanoparticle-based short interfering RNA drug for the treatment of polyneuropathies induced by hereditary…”
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Expression kinetics of nucleoside-modified mRNA delivered in lipid nanoparticles to mice by various routes by Pardi, Norbert, Tuyishime, Steven, Muramatsu, Hiromi, Kariko, Katalin, Mui, Barbara L., Tam, Ying K., Madden, Thomas D., Hope, Michael J., Weissman, Drew

“…In recent years, in vitro transcribed messenger RNA (mRNA) has emerged as a potential therapeutic platform. To fulfill its promise, effective delivery of mRNA…”
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Nucleoside-modified mRNA immunization elicits influenza virus hemagglutinin stalk-specific antibodies by Pardi, Norbert, Parkhouse, Kaela, Kirkpatrick, Ericka, McMahon, Meagan, Zost, Seth J., Mui, Barbara L., Tam, Ying K., Karikó, Katalin, Barbosa, Christopher J., Madden, Thomas D., Hope, Michael J., Krammer, Florian, Hensley, Scott E., Weissman, Drew

“…Currently available influenza virus vaccines have inadequate effectiveness and are reformulated annually due to viral antigenic drift. Thus, development of a…”
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Administration of nucleoside-modified mRNA encoding broadly neutralizing antibody protects humanized mice from HIV-1 challenge by Pardi, Norbert, Secreto, Anthony J., Shan, Xiaochuan, Debonera, Fotini, Glover, Joshua, Yi, Yanjie, Muramatsu, Hiromi, Ni, Houping, Mui, Barbara L., Tam, Ying K., Shaheen, Farida, Collman, Ronald G., Karikó, Katalin, Danet-Desnoyers, Gwenn A., Madden, Thomas D., Hope, Michael J., Weissman, Drew

“…Monoclonal antibodies are one of the fastest growing classes of pharmaceutical products, however, their potential is limited by the high cost of development…”
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PECAM-1 directed re-targeting of exogenous mRNA providing two orders of magnitude enhancement of vascular delivery and expression in lungs independent of apolipoprotein E-mediated uptake by Parhiz, Hamideh, Shuvaev, Vladimir V., Pardi, Norbert, Khoshnejad, Makan, Kiseleva, Raisa Yu, Brenner, Jacob S., Uhler, Thomas, Tuyishime, Steven, Mui, Barbara L., Tam, Ying K., Madden, Thomas D., Hope, Michael J., Weissman, Drew, Muzykantov, Vladimir R.

“…Systemic administration of lipid nanoparticle (LNP)-encapsulated messenger RNA (mRNA) leads predominantly to hepatic uptake and expression. Here, we conjugated…”
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Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo by Jayaraman, Muthusamy, Ansell, Steven M., Mui, Barbara L., Tam, Ying K., Chen, Jianxin, Du, Xinyao, Butler, David, Eltepu, Laxman, Matsuda, Shigeo, Narayanannair, Jayaprakash K., Rajeev, Kallanthottathil G., Hafez, Ismail M., Akinc, Akin, Maier, Martin A., Tracy, Mark A., Cullis, Pieter R., Madden, Thomas D., Manoharan, Muthiah, Hope, Michael J.

“…Special (lipid) delivery: The role of the ionizable lipid pKa in the in vivo delivery of siRNA by lipid nanoparticles has been studied with a large number of…”
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mRNA mediates passive vaccination against infectious agents, toxins, and tumors by Thran, Moritz, Mukherjee, Jean, Pönisch, Marion, Fiedler, Katja, Thess, Andreas, Mui, Barbara L, Hope, Michael J, Tam, Ying K, Horscroft, Nigel, Heidenreich, Regina, Fotin‐Mleczek, Mariola, Shoemaker, Charles B, Schlake, Thomas

“…The delivery of genetic information has emerged as a valid therapeutic approach. Various reports have demonstrated that mRNA, besides its remarkable potential…”
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Lipid Nanoparticles Containing siRNA Synthesized by Microfluidic Mixing Exhibit an Electron-Dense Nanostructured Core by Leung, Alex K. K, Hafez, Ismail M, Baoukina, Svetlana, Belliveau, Nathan M, Zhigaltsev, Igor V, Afshinmanesh, Elham, Tieleman, D. Peter, Hansen, Carl L, Hope, Michael J, Cullis, Pieter R

“…Lipid nanoparticles (LNP) containing ionizable cationic lipids are the leading systems for enabling therapeutic applications of siRNA; however, the structure…”
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Rational design of cationic lipids for siRNA delivery by Semple, Sean C, Akinc, Akin, Chen, Jianxin, Sandhu, Ammen P, Mui, Barbara L, Cho, Connie K, Sah, Dinah W Y, Stebbing, Derrick, Crosley, Erin J, Yaworski, Ed, Hafez, Ismail M, Dorkin, J Robert, Qin, June, Lam, Kieu, Rajeev, Kallanthottathil G, Wong, Kim F, Jeffs, Lloyd B, Nechev, Lubomir, Eisenhardt, Merete L, Jayaraman, Muthusamy, Kazem, Mikameh, Maier, Martin A, Srinivasulu, Masuna, Weinstein, Michael J, Chen, Qingmin, Alvarez, Rene, Barros, Scott A, De, Soma, Klimuk, Sandra K, Borland, Todd, Kosovrasti, Verbena, Cantley, William L, Tam, Ying K, Manoharan, Muthiah, Ciufolini, Marco A, Tracy, Mark A, de Fougerolles, Antonin, MacLachlan, Ian, Cullis, Pieter R, Madden, Thomas D, Hope, Michael J

“…We adopted a rational approach to design cationic lipids for use in formulations to deliver small interfering RNA (siRNA). Starting with the ionizable cationic…”
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Unmodified mRNA in LNPs constitutes a competitive technology for prophylactic vaccines by Lutz, Johannes, Lazzaro, Sandra, Habbeddine, Mohamed, Schmidt, Kim Ellen, Baumhof, Patrick, Mui, Barbara L., Tam, Ying K., Madden, Thomas D., Hope, Michael J., Heidenreich, Regina, Fotin-Mleczek, Mariola

“…mRNA represents a promising new vaccine technology platform with high flexibility in regard to development and production. Here, we demonstrate that vaccines…”
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Nucleoside-modified VEGFC mRNA induces organ-specific lymphatic growth and reverses experimental lymphedema by Szőke, Dániel, Kovács, Gábor, Kemecsei, Éva, Bálint, László, Szoták-Ajtay, Kitti, Aradi, Petra, Styevkóné Dinnyés, Andrea, Mui, Barbara L., Tam, Ying K., Madden, Thomas D., Karikó, Katalin, Kataru, Raghu P., Hope, Michael J., Weissman, Drew, Mehrara, Babak J., Pardi, Norbert, Jakus, Zoltán

“…Lack or dysfunction of the lymphatics leads to secondary lymphedema formation that seriously reduces the function of the affected organs and results in…”
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Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles by Mui, Barbara L, Tam, Ying K, Jayaraman, Muthusamy, Ansell, Steven M, Du, Xinyao, Tam, Yuen Yi C, Lin, Paulo JC, Chen, Sam, Narayanannair, Jayaprakash K, Rajeev, Kallanthottathil G, Manoharan, Muthiah, Akinc, Akin, Maier, Martin A, Cullis, Pieter, Madden, Thomas D, Hope, Michael J

“…Lipid nanoparticles (LNPs) encapsulating short interfering RNAs that target hepatic genes are advancing through clinical trials, and early results indicate the…”
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Characterization of HIV-1 Nucleoside-Modified mRNA Vaccines in Rabbits and Rhesus Macaques by Pardi, Norbert, LaBranche, Celia C., Ferrari, Guido, Cain, Derek W., Tombácz, István, Parks, Robert J., Muramatsu, Hiromi, Mui, Barbara L., Tam, Ying K., Karikó, Katalin, Polacino, Patricia, Barbosa, Christopher J., Madden, Thomas D., Hope, Michael J., Haynes, Barton F., Montefiori, David C., Hu, Shiu-Lok, Weissman, Drew

“…Despite the enormous effort in the development of effective vaccines against HIV-1, no vaccine candidate has elicited broadly neutralizing antibodies in…”
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Influence of Cationic Lipid Composition on Gene Silencing Properties of Lipid Nanoparticle Formulations of siRNA in Antigen-Presenting Cells by Basha, Genc, Novobrantseva, Tatiana I, Rosin, Nicole, Tam, Yuen Yi C, Hafez, Ismail M, Wong, Matthew K, Sugo, Tsukasa, Ruda, Vera M, Qin, June, Klebanov, Boris, Ciufolini, Marco, Akinc, Akin, Tam, Ying K, Hope, Michael J, Cullis, Pieter R

“…Lipid nanoparticles (LNPs) are currently the most effective in vivo delivery systems for silencing target genes in hepatocytes employing small interfering RNA…”
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Lipid Nanoparticle Systems for Enabling Gene Therapies by Cullis, Pieter R., Hope, Michael J.

“…Genetic drugs such as small interfering RNA (siRNA), mRNA, or plasmid DNA provide potential gene therapies to treat most diseases by silencing pathological…”
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Immunogenicity and rapid blood clearance of liposomes containing polyethylene glycol-lipid conjugates and nucleic Acid by Semple, Sean C, Harasym, Troy O, Clow, Kathy A, Ansell, Steven M, Klimuk, Sandra K, Hope, Michael J

“…Polyethylene glycol (PEG) is used widely in the pharmaceutical industry to improve the pharmacokinetics and reduce the immunogenicity of therapeutic and…”
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Zika virus protection by a single low-dose nucleoside-modified mRNA vaccination by Pardi, Norbert, Hogan, Michael J., Pelc, Rebecca S., Muramatsu, Hiromi, Andersen, Hanne, DeMaso, Christina R., Dowd, Kimberly A., Sutherland, Laura L., Scearce, Richard M., Parks, Robert, Wagner, Wendeline, Granados, Alex, Greenhouse, Jack, Walker, Michelle, Willis, Elinor, Yu, Jae-Sung, McGee, Charles E., Sempowski, Gregory D., Mui, Barbara L., Tam, Ying K., Huang, Yan-Jang, Vanlandingham, Dana, Holmes, Veronica M., Balachandran, Harikrishnan, Sahu, Sujata, Lifton, Michelle, Higgs, Stephen, Hensley, Scott E., Madden, Thomas D., Hope, Michael J., Karikó, Katalin, Santra, Sampa, Graham, Barney S., Lewis, Mark G., Pierson, Theodore C., Haynes, Barton F., Weissman, Drew

“…A single, low-dose intradermal immunization with lipid-nanoparticle-encapsulated nucleoside-modified mRNA encoding the pre-membrane and envelope glycoproteins…”
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Nucleoside-modified mRNA vaccines induce potent T follicular helper and germinal center B cell responses by Pardi, Norbert, Hogan, Michael J, Naradikian, Martin S, Parkhouse, Kaela, Cain, Derek W, Jones, Letitia, Moody, M Anthony, Verkerke, Hans P, Myles, Arpita, Willis, Elinor, LaBranche, Celia C, Montefiori, David C, Lobby, Jenna L, Saunders, Kevin O, Liao, Hua-Xin, Korber, Bette T, Sutherland, Laura L, Scearce, Richard M, Hraber, Peter T, Tombácz, István, Muramatsu, Hiromi, Ni, Houping, Balikov, Daniel A, Li, Charles, Mui, Barbara L, Tam, Ying K, Krammer, Florian, Karikó, Katalin, Polacino, Patricia, Eisenlohr, Laurence C, Madden, Thomas D, Hope, Michael J, Lewis, Mark G, Lee, Kelly K, Hu, Shiu-Lok, Hensley, Scott E, Cancro, Michael P, Haynes, Barton F, Weissman, Drew

“…T follicular helper (Tfh) cells are required to develop germinal center (GC) responses and drive immunoglobulin class switch, affinity maturation, and…”
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Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics by Maier, Martin A, Jayaraman, Muthusamy, Matsuda, Shigeo, Liu, Ju, Barros, Scott, Querbes, William, Tam, Ying K, Ansell, Steven M, Kumar, Varun, Qin, June, Zhang, Xuemei, Wang, Qianfan, Panesar, Sue, Hutabarat, Renta, Carioto, Mary, Hettinger, Julia, Kandasamy, Pachamuthu, Butler, David, Rajeev, Kallanthottathil G, Pang, Bo, Charisse, Klaus, Fitzgerald, Kevin, Mui, Barbara L, Du, Xinyao, Cullis, Pieter, Madden, Thomas D, Hope, Michael J, Manoharan, Muthiah, Akinc, Akin

“…In recent years, RNA interference (RNAi) therapeutics, most notably with lipid nanoparticle-based delivery systems, have advanced into human clinical trials…”
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