Targeted mRNA Therapy for Ornithine Transcarbamylase Deficiency

We describe a novel, two-nanoparticle mRNA delivery system and show that it is highly effective as a means of intracellular enzyme replacement therapy (i-ERT) using a murine model of ornithine transcarbamylase deficiency (OTCD). Our Hybrid mRNA Technology delivery system (HMT) comprises an inert lip...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Prieve, Mary G., Harvie, Pierrot, Monahan, Sean D., Roy, Debashish, Li, Allen G., Blevins, Teri L., Paschal, Amber E., Waldheim, Matt, Bell, Eric C., Galperin, Anna, Ella-Menye, Jean-Rene, Houston, Michael E.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5910669/
https://ncbi.nlm.nih.gov/pubmed/29433939
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.12.024
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