Systematic evaluation of 2′-Fluoro modified chimeric antisense oligonucleotide-mediated exon skipping in vitro

S Chen, BT Le, M Chakravarthy, TR Kosbar… - Scientific Reports, 2019 - nature.com
Antisense oligonucleotide (AO)-mediated splice modulation has been established as a
therapeutic approach for tackling genetic diseases. Recently, Exondys51, a drug that aims to …

Compatibility of 5-ethynyl-2′ F-ANA UTP with in vitro selection for the generation of base-modified, nuclease resistant aptamers

F Levi-Acobas, A Katolik, P Röthlisberger… - Organic & …, 2019 - pubs.rsc.org
A modified nucleoside triphosphate bearing two modifications based on a 2′-deoxy-2′-
fluoro-arabinofuranose sugar and a uracil nucleobase equipped with a C5-ethynyl moiety (5 …

Strong inhibitory effects of antisense probes on gene expression through ultrafast RNA photocrosslinking

K Fujimoto, H Yang‐Chun… - Chemistry–An Asian …, 2019 - Wiley Online Library
We have reported the photochemical regulation of the intracellular antisense effect of
antisense probes containing a photo‐responsive artificial nucleic acid, 3 …

Enhancement of exon skipping activity by reduction in the secondary structure content of LNA-based splice-switching oligonucleotides

T Shimo, K Tachibana, Y Kawawaki… - Chemical …, 2019 - pubs.rsc.org
PAGE and UV melting analysis revealed that longer LNA-based splice-switching
oligonucleotides (SSOs) formed secondary structures by themselves, reducing their effective …

BACE1 inhibition using 2'-OMePS steric blocking antisense oligonucleotides

M Chakravarthy, RN Veedu - Genes, 2019 - mdpi.com
Amyloid beta-peptide is produced by the cleavage of amyloid precursor protein by two
secretases, a β-secretase, beta-site amyloid precursor protein cleaving enzyme 1 (BACE1) …

Kinetic analysis of N-alkylaryl carboxamide hexitol nucleotides as substrates for evolved polymerases

M Renders, S Dumbre, M Abramov… - Nucleic Acids …, 2019 - academic.oup.com
Abstract Six 1′, 5′-anhydrohexitol uridine triphosphates were synthesized with aromatic
substitutions appended via a carboxamide linker to the 5-position of their bases. An …