Chemistry, structure and function of approved oligonucleotide therapeutics

M Egli, M Manoharan - Nucleic Acids Research, 2023 - academic.oup.com
Eighteen nucleic acid therapeutics have been approved for treatment of various diseases in
the last 25 years. Their modes of action include antisense oligonucleotides (ASOs), splice …

The medicinal chemistry of therapeutic oligonucleotides

WB Wan, PP Seth - Journal of medicinal chemistry, 2016 - ACS Publications
Oligonucleotide-based therapeutics have made rapid progress in the clinic for treatment of a
variety of disease indications. Unmodified oligonucleotides are polyanionic macromolecules …

Advancement of the emerging field of RNA nanotechnology

D Jasinski, F Haque, DW Binzel, P Guo - ACS nano, 2017 - ACS Publications
The field of RNA nanotechnology has advanced rapidly during the past decade. A variety of
programmable RNA nanoparticles with defined shape, size, and stoichiometry have been …

A field guide to foldamers

DJ Hill, MJ Mio, RB Prince, TS Hughes… - Chemical …, 2001 - ACS Publications
The breadth of structure and function displayed by the molecules of biology is remarkable.
Considering that there are only three major biopolymer backbones (proteins, ribonucleic …

Antisense technologies: improvement through novel chemical modifications

J Kurreck - European journal of biochemistry, 2003 - Wiley Online Library
Antisense agents are valuable tools to inhibit the expression of a target gene in a sequence‐
specific manner, and may be used for functional genomics, target validation and therapeutic …

Sensitive and specific detection of microRNAs by northern blot analysis using LNA-modified oligonucleotide probes

A Válóczi, C Hornyik, N Varga, J Burgyán… - Nucleic acids …, 2004 - academic.oup.com
We describe here a new method for highly efficient detection of microRNAs by northern blot
analysis using LNA (locked nucleic acid)-modified oligonucleotides. In order to exploit the …

Locked nucleic acid (LNA): fine-tuning the recognition of DNA and RNA

DA Braasch, DR Corey - Chemistry & biology, 2001 - cell.com
Locked nucleic acid is an RNA derivative in which the ribose ring is constrained by a
methylene linkage between the 2′-oxygen and the 4′-carbon. This conformation …

Design of antisense oligonucleotides stabilized by locked nucleic acids

J Kurreck, E Wyszko, C Gillen… - Nucleic acids …, 2002 - academic.oup.com
The design of antisense oligonucleotides containing locked nucleic acids (LNA) was
optimized and compared to intensively studied DNA oligonucleotides, phosphorothioates …

Locked nucleic acid (LNA) mediated improvements in siRNA stability and functionality

J Elmén, H Thonberg, K Ljungberg… - Nucleic acids …, 2005 - academic.oup.com
Therapeutic application of the recently discovered small interfering RNA (siRNA) gene
silencing phenomenon will be dependent on improvements in molecule bio-stability …

LNA: a versatile tool for therapeutics and genomics

M Petersen, J Wengel - Trends in biotechnology, 2003 - cell.com
Locked nucleic acid (LNA) is a nucleic acid analogue that displays unprecedented
hybridization affinity towards complementary DNA and RNA. Structural studies have shown …