Development of an mRNA replacement therapy for phenylketonuria by Carlos G. Perez-Garcia, Ramon Diaz-Trelles, Jerel Boyd Vega, Yanjie Bao, Marciano Sablad, Patty Limphong, Simon Chikamatsu, Hailong Yu, Wendy Taylor, Priya P. Karmali, Kiyoshi Tachikawa, Padmanabh Chivukula

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Selective suppression of polyglutamine-expanded protein by lipid nanoparticle-delivered siRNA targeting CAG expansions in the mouse CNS by Tomoki Hirunagi, Kentaro Sahashi, Kiyoshi Tachikawa, Angel I. Leu, Michelle Nguyen, Rajesh Mukthavaram, Priya P. Karmali, Padmanabh Chivukula, Genki Tohnai, Madoka Iida, Kazunari Onodera, Manabu Ohyama, Yohei Okada, Hideyuki Okano, Masahisa Katsuno

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Downregulation of hepatic lipopolysaccharide binding protein improves lipogenesis-induced liver lipid accumulation by Jessica Latorre, Ramon Díaz-Trelles, Ferran Comas, Aleix Gavaldà-Navarro, Edward Milbank, Nathalia Dragano, Samantha Morón-Ros, Rajesh Mukthavaram, Francisco Ortega, Anna Castells-Nobau, Núria Oliveras-Cañellas, Wifredo Ricart, Priya P. Karmali, Kiyoshi Tachikawa, Pad Chivukula, Francesc Villarroya, Miguel López, Marta Giralt, José Manuel Fernández-Real, José María Moreno-Navarrete

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