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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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| 出版年: | Mol Ther |
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| 主要な著者: | , , , , , , , , , , , |
| フォーマット: | Artigo |
| 言語: | Inglês |
| 出版事項: |
American Society of Gene & Cell Therapy
2018
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| 主題: | |
| オンライン･アクセス: | 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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