The beauty of symmetry: siRNA phosphorodithioate modifications reduce stereocomplexity, ease analysis, and can improve in vivo potency

The beauty of symmetry: siRNA phosphorodithioate modifications reduce stereocomplexity, ease analysis, and can improve in vivo potency

Phosphorothioates (PSs) can be essential in stabilizing therapeutic oligonucleotides against enzymatic degradation. However, unless synthesis is performed with stereodefined amidites, each PS introduces a chemically undefined stereocenter, resulting in 2n unique molecules in the final product and af...

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Bibliographic Details
Main Authors: Sophie Schöllkopf, Stefan Rathjen, Micaela Graglia, Nina Was, Eliot Morrison, Adrien Weingärtner, Lucas Bethge, Judith Hauptmann, Marie Wikström Lindholm
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Oligonucleotides: Therapies and Applications
siRNA conjugate
GalNAc
phosphorothioate
phosphorodithioate
stereopure
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253124002233

Internet

http://www.sciencedirect.com/science/article/pii/S2162253124002233

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