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Lipid nanoparticle delivers phenylalanine ammonia lyase mRNA to the liver leading to catabolism and clearance of phenylalanine in a phenylketonuria mouse model by Ramon Diaz-Trelles, Sharon Lee, Kristen Kuakini, Jenny Park, Adrian Dukanovic, Jose A. Gonzalez, Thanhchau Dam, Jae Heon Kim, Jerel Boyd Vega, Marciano Sablad, Priya P. Karmali, Kiyoshi Tachikawa, Padmanabh Chivukula
Published 2022-09-01
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Restoring ornithine transcarbamylase (OTC) activity in an OTC‐deficient mouse model using LUNAR‐OTC mRNA by Hailong Yu, Edward Brewer, Michael Shields, Michael Crowder, Cristiano Sacchetti, Benchawanna Soontornniyomkij, Dandan Dou, Brenda Clemente, Marciano Sablad, Padmanabh Chivukula, Steve Hughes, Scott Roberts, Kumar Rajappan, Steve Tannis, Rose Sekulovich, Suezanne Parker, Pattraranee Limphong
Published 2022-06-01
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