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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...

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Detalles Bibliográficos
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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
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