Article
Structurally constrained phosphonate internucleotide linkage impacts oligonucleotide-enzyme interaction, and modulates siRNA activity and allele specificity.
Nucleic acids research - 2 Dec 2021
Yamada Ken, Hildebrand Samuel, Davis Sarah M, Miller Rachael, Conroy Faith, Sapp Ellen, Caiazzi Jillian, Alterman Julia F, Roux Loic, Echeverria Dimas, Hassler Matthew R, Pfister Edith L, DiFiglia Marian, Aronin Neil, Khvorova Anastasia
Abstract excerpt
Oligonucleotides is an emerging class of chemically-distinct therapeutic modalities, where extensive chemical modifications are fundamental for their clinical applications. Inter-nucleotide backbones are critical to the behaviour of therapeutic oligonucleotides, but clinically explored backbone analogues are, effectively, limited to phosphorothioates. Here, we describe the synthesis and bio-functional...
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