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Intermittent lipid nanoparticle mRNA administration prevents cortical dysmyelination associated with arginase deficiency by Suhail Khoja, Xiao-Bo Liu, Brian Truong, Matthew Nitzahn, Jenna Lambert, Adam Eliav, Eram Nasser, Emma Randolph, Kristine E. Burke, Rebecca White, Xuling Zhu, Paolo G.V. Martini, Itzhak Nissim, Stephen D. Cederbaum, Gerald S. Lipshutz
Published 2022-06-01
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Gene therapy for guanidinoacetate methyltransferase deficiency restores cerebral and myocardial creatine while resolving behavioral abnormalities by Suhail Khoja, Jenna Lambert, Matthew Nitzahn, Adam Eliav, YuChen Zhang, Mikayla Tamboline, Colleen T. Le, Eram Nasser, Yunfeng Li, Puja Patel, Irina Zhuravka, Lindsay M. Lueptow, Ilona Tkachyova, Shili Xu, Itzhak Nissim, Andreas Schulze, Gerald S. Lipshutz
Published 2022-06-01
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