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Integrated Assessment of the Clinical Performance of GalNAc(3)-Conjugated 2′-O-Methoxyethyl Chimeric Antisense Oligonucleotides: I. Human Volunteer Experience
Advances in medicinal chemistry have produced new chemical classes of antisense oligonucleotides (ASOs) with enhanced therapeutic properties. Conjugation of the triantennary N-acetylgalactosamine (GalNAc(3)) moiety to the extensively characterized phosphorothioate (PS)-modified 2′-O-methoxyethyl (2′...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Mary Ann Liebert, Inc., publishers
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6386089/ https://www.ncbi.nlm.nih.gov/pubmed/30570431 http://dx.doi.org/10.1089/nat.2018.0753 |