Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates
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.
Published in American journal of human genetics (04.04.2019)
Published in American journal of human genetics (04.04.2019)
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Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia
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.
Published in Cell reports (Cambridge) (19.12.2017)
Published in Cell reports (Cambridge) (19.12.2017)
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mRNA Therapy Improves Metabolic and Behavioral Abnormalities in a Murine Model of Citrin Deficiency
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.
Published in Molecular therapy (03.07.2019)
Published in Molecular therapy (03.07.2019)
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Journal Article
Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemia
An, Ding, Frassetto, Andrea, Jacquinet, Eric, Eybye, Marianne, Milano, Joseph, DeAntonis, Christine, Nguyen, Vi, Laureano, Rodrigo, Milton, Jaclyn, Sabnis, Staci, Lukacs, Christine M., Guey, Lin T.
Published in EBioMedicine (01.07.2019)
Published in EBioMedicine (01.07.2019)
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Accelerated Blood Clearance of Lipid Nanoparticles Entails a Biphasic Humoral Response of B-1 Followed by B-2 Lymphocytes to Distinct Antigenic Moieties
Besin, Gilles, Milton, Jaclyn, Sabnis, Staci, Howell, Rebecca, Mihai, Cosmin, Burke, Kristine, Benenato, Kerry E, Stanton, Matthew, Smith, Peter, Senn, Joseph, Hoge, Stephen
Published in ImmunoHorizons (01.07.2019)
Published in ImmunoHorizons (01.07.2019)
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Discovery of a Novel Amino Lipid That Improves Lipid Nanoparticle Performance through Specific Interactions with mRNA
Cornebise, Mark, Narayanan, Elisabeth, Xia, Yan, Acosta, Edward, Ci, Lei, Koch, Hillary, Milton, Jaclyn, Sabnis, Staci, Salerno, Timothy, Benenato, Kerry E.
Published in Advanced functional materials (01.02.2022)
Published in Advanced functional materials (01.02.2022)
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Systemic messenger RNA as an etiological treatment for acute intermittent porphyria
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
Published in Nature medicine (01.12.2018)
Published in Nature medicine (01.12.2018)
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Journal Article
A Novel Amino Lipid Series for mRNA Delivery: Improved Endosomal Escape and Sustained Pharmacology and Safety in Non-human Primates
Sabnis, Staci, Kumarasinghe, E. Sathyajith, Salerno, Timothy, Mihai, Cosmin, Ketova, Tatiana, Senn, Joseph J., Lynn, Andy, Bulychev, Alex, McFadyen, Iain, Chan, Joyce, Almarsson, Örn, Stanton, Matthew G., Benenato, Kerry E.
Published in Molecular therapy (06.06.2018)
Published in Molecular therapy (06.06.2018)
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Journal Article
Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia
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.
Published in Cell reports (Cambridge) (28.08.2018)
Published in Cell reports (Cambridge) (28.08.2018)
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Journal Article
Efficacy of systemic messenger RNA therapy to treat and prevent porphyria attacks in animal models of acute intermittent porphyria
Jiang, Lei, Berraondo, Pedro, Guey, Lin, Sampedro, Ana, Frassetto, Andrea, Benenato, Kerry, Kalariya, Mayur, Sabnis, Staci, Salerno, Timothy, Kumarasinghe, Ellalahewage, Butcher, William, Lukacs, Christine, Ávila, Matías A., Fontanellas, Antonio, Martini, Paolo
Published in Molecular genetics and metabolism (01.02.2018)
Published in Molecular genetics and metabolism (01.02.2018)
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Long-term efficacy and safety of mRNA therapy in two murine models of methylmalonic acidemiaResearch in context
Ding An, Andrea Frassetto, Eric Jacquinet, Marianne Eybye, Joseph Milano, Christine DeAntonis, Vi Nguyen, Rodrigo Laureano, Jaclyn Milton, Staci Sabnis, Christine M. Lukacs, Lin T. Guey
Published in EBioMedicine (01.07.2019)
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Published in EBioMedicine (01.07.2019)
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Year of Publication 29.06.2023
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Year of Publication 29.06.2023
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Year of Publication 25.08.2022
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Year of Publication 29.07.2022
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Year of Publication 28.07.2022
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Year of Publication 25.07.2022
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Year of Publication 04.07.2022
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Year of Publication 30.06.2022
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Year of Publication 30.06.2022
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