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收藏mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease
Cao, Jingsong; Choi, Minjung; Guadagnin, Eleonora; Soty, Maud; Silva, Marine; Verzieux, Vincent; Weisser, Edward; Markel, Arianna; Zhuo, Jenny; Liang, Shi; Yin, Ling; Frassetto, Andrea; Graham, Anne-Renee; Burke, Kristine; Ketova, Tatiana; Mihai, Cosmin; Zalinger, Zach; Levy, Becca; Besin, Gilles; Wolfrom, Meredith; Tran, Barbara; Tunkey, Christopher; Owen, Erik; Sarkis, Joe; Dousis, Athanasios; Presnyak, Vladimir; Pepin, Christopher; Zheng, Wei; Ci, Lei; Hard, Marjie; Miracco, Edward; Rice, Lisa; Vi Nguyen; Zimmer, Mike; Rajarajacholan, Uma; Finn, Patrick F.; Mithieux, Gilles; Rajas, Fabienne; Martini, Paolo G., V; Giangrande, Paloma H.
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收藏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.
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收藏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.
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收藏Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia (vol 21, pg 3548, 2017)
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.
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收藏Systemic mRNA therapy for the treatment of Fabry disease: Preclinical studies in GLA-deficient mice and rats, and wild-typenon-human primates
Zhu, Xuling; Yin, Ling; Zhuo, Jenny; Miller, James J.; Siddiqui, Summar; Theisen, Matt; Levy, Becca; Howell, Rebecca; Presnyak, Vladimir; Beltrame, Angela; Murphy, Carly; Frassetto, Andrea; Milano, Joe; Lynn, Andy; Besin, Gilles; Benenato, Kerry; Lukacs, Christine; Dahms, Nancy; Guey, Lin; Martini, Paolo
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收藏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.
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