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′...

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Publicat a:Nucleic Acid Ther
Autors principals: Crooke, Stanley T., Baker, Brenda F., Xia, Shuting, Yu, Rosie Z., Viney, Nicholas J., Wang, Yanfeng, Tsimikas, Sotirios, Geary, Richard S.
Format: Artigo
Idioma:Inglês
Publicat: Mary Ann Liebert, Inc., publishers 2019
Matèries:
Original Articles
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6386089/
https://ncbi.nlm.nih.gov/pubmed/30570431
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/nat.2018.0753
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