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Ligand conjugate SAR and enhanced delivery in NHP
N-Acetylgalactosamine (GalNAc) conjugated short interfering RNAs (siRNAs) are a leading RNA interference (RNAi) platform allowing targeted inhibition of disease-causing genes in hepatocytes. More than a decade of development has recently resulted in the first approvals for this class of drugs. While...
Autores principales: | Holland, Richard James, Lam, Kieu, Ye, Xin, Martin, Alan D., Wood, Mark C., Palmer, Lorne, Fraser, David, McClintock, Kevin, Majeski, Sara, Jarosz, Agnes, Lee, Amy C.H., Thi, Emily P., Judge, Adam, Heyes, James |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Society of Gene & Cell Therapy
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8531135/ https://www.ncbi.nlm.nih.gov/pubmed/34091052 http://dx.doi.org/10.1016/j.ymthe.2021.06.002 |
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