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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Detalles Bibliográficos
Autores principales: Weingärtner, Adrien, Bethge, Lucas, Weiss, Lisa, Sternberger, Maria, Lindholm, Marie Wikstrom
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7321783/
https://www.ncbi.nlm.nih.gov/pubmed/32590173
http://dx.doi.org/10.1016/j.omtn.2020.05.026

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7321783/
https://www.ncbi.nlm.nih.gov/pubmed/32590173
http://dx.doi.org/10.1016/j.omtn.2020.05.026

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