Antisense oligonucleotide-mediated splice switching: potential therapeutic approach for cancer mitigation

P Raguraman, AA Balachandran, S Chen… - Cancers, 2021 - mdpi.com
Simple Summary Splicing is an important mechanism by which precursor mRNA is modified
into mature mRNA. This splicing plays a major role in the generation of different proteins …

Thiomorpholino oligonucleotides as a robust class of next generation platforms for alternate mRNA splicing

BT Le, S Paul, K Jastrzebska… - Proceedings of the …, 2022 - National Acad Sciences
Recent advances in drug development have seen numerous successful clinical translations
using synthetic antisense oligonucleotides (ASOs). However, major obstacles, such as …

Synthesis of a convertible linker containing a disulfide group for oligonucleotide functionalization

A Pontarelli, JT Liu, H Movasat, S Ménard, JK Oh… - Organic …, 2022 - ACS Publications
The synthesis and incorporation of a tosylated phosphoramidite linker containing a disulfide
bond is described. Incorporation of the linker into short DNA and RNA oligomers proceeded …

Lipid-linked nucleoside triphosphates for enzymatic synthesis of hydrophobic oligonucleotides with enhanced membrane anchoring efficiency

D Kodr, E Kužmová, R Pohl, T Kraus, M Hocek - Chemical Science, 2023 - pubs.rsc.org
We designed and synthesized a series of 2′-deoxyribonucleoside triphosphates (dNTPs)
bearing various lipid moieties. Fatty acid-and cholesterol-modified dNTPs proved to be …

Antisense Oligonucleotide Modified with Disulfide Units Induces Efficient Exon Skipping in mdx Myotubes through Enhanced Membrane Permeability and Nucleus …

H Hiraoka, Z Shu, B Tri Le, K Masuda… - …, 2021 - Wiley Online Library
We have found that antisense oligonucleotides and siRNA molecules modified with repeat
structures of disulfide units can be directly introduced into the cytoplasm and exhibit a …

Splice-Switching Antisense Oligonucleotides Targeting Extra-and Intracellular Domains of Epidermal Growth Factor Receptor in Cancer Cells

AA Balachandran, P Raguraman, K Rahimizadeh… - Biomedicines, 2023 - mdpi.com
Cancer is one of the leading causes of death globally. Epidermal growth factor receptor is
one of the proteins involved in cancer cell proliferation, differentiation, and invasion …

Phosphorothioate modification improves exon-skipping of antisense oligonucleotides based on sulfonyl phosphoramidates in mdx mouse myotubes

Y Su, P Raguraman, RN Veedu… - Organic & Biomolecular …, 2022 - pubs.rsc.org
2′-O-Methyl (2′-OMe) antisense oligonucleotides (AOs) possessing a various number of
4-(trimethylammonio) butylsulfonyl or tosyl phosphoramidates (N+ and Ts-modifications …

Intracellular Delivery of Antisense Oligonucleotides by Tri‐Branched Cyclic Disulfide Units

F Lyu, H Hakariya, H Hiraoka, Z Li… - …, 2024 - Wiley Online Library
Therapeutic oligonucleotides, such as antisense DNA, show promise in treating previously
untreatable diseases. However, their applications are still hindered by the poor membrane …

Oligonucleotides containing 2′-O-methyl-5-(1-phenyl-1, 2, 3-triazol-4-yl) uridines demonstrate increased affinity for RNA and induce exon-skipping in vitro

AG Molina, P Raguraman, L Delcomyn… - Bioorganic & Medicinal …, 2022 - Elsevier
The nucleotide monomer containing the 1-phenyl-1, 2, 3-triazole group attached to the 5-
position of 2′-O-methyluridine is hereby presented together with a derivative further …

Oligodeoxynucleotides Modified with 2′-O-(Cysteinylaminobutyl)carbamoylethylribothymidine Residues for Native Chemical Ligation with Peptide at Internal …

Y Masaki, A Maruyama, K Yoshida, T Tomori… - Bioconjugate …, 2022 - ACS Publications
We used native chemical ligation (NCL) to synthesize a 2′-O-{N-[N-(S-tert-
butylthiocysteinyl) aminobutyl] carbamoylethyl}(CysBCE) ribothymidine-derived …