7′,5′-alpha-bicyclo-DNA: new chemistry for oligonucleotide exon splicing modulation therapy

Abstract Antisense oligonucleotides are small pieces of modified DNA or RNA, which offer therapeutic potential for many diseases. We report on the synthesis of 7′,5′-α-bc-DNA phosphoramidite building blocks, bearing the A, G, T and MeC nucleobases. Solid-phase synthesis was performed to construct fi...

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Veröffentlicht in:	Nucleic acids research 2021-12, Vol.49 (21), p.12089-12105
Hauptverfasser:	Evéquoz, Damien, Verhaart, Ingrid E C, van de Vijver, Davy, Renner, Wolfgang, Aartsma-Rus, Annemieke, Leumann, Christian J
Format:	Artikel
Sprache:	eng
Schlagworte:	Animals Cell Line Chemical Biology and Nucleic Acid Chemistry DNA - therapeutic use Genetic Therapy - methods Male Mice Muscular Atrophy, Spinal - therapy Muscular Dystrophy, Duchenne - therapy Oligonucleotides - chemistry Oligonucleotides - therapeutic use
Online-Zugang:	Volltext
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