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Less Is More: Novel Hepatocyte-Targeted siRNA Conjugates for Treatment of Liver-Related Disorders
N-acetyl-galactosamine (GalNAc) conjugation enhances liver specificity for therapeutic oligonucleotides. Here we report on a novel design with improved activity and stability compared with a triantennary design. We applied a versatile monovalent serinol-GalNAc conjugation strategy. First, 1–4 serial...
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| Publicat a: | Mol Ther Nucleic Acids |
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| Autors principals: | , , , , |
| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
American Society of Gene & Cell Therapy
2020
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7321783/ https://ncbi.nlm.nih.gov/pubmed/32590173 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2020.05.026 |
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