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 …

[HTML][HTML] Emerging concepts of miRNA therapeutics: from cells to clinic

C Diener, A Keller, E Meese - Trends in Genetics, 2022 - cell.com
MicroRNAs (miRNAs) are very powerful genetic regulators, as evidenced by the fact that a
single miRNA can direct entire cellular pathways via interacting with a broad spectrum of …

Translational applications of hydrogels

S Correa, AK Grosskopf, H Lopez Hernandez… - Chemical …, 2021 - ACS Publications
Advances in hydrogel technology have unlocked unique and valuable capabilities that are
being applied to a diverse set of translational applications. Hydrogels perform functions …

Advances in oligonucleotide drug delivery

TC Roberts, R Langer, MJA Wood - Nature reviews Drug discovery, 2020 - nature.com
Oligonucleotides can be used to modulate gene expression via a range of processes
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …

Delivery of oligonucleotide‐based therapeutics: challenges and opportunities

SM Hammond, A Aartsma‐Rus, S Alves… - EMBO molecular …, 2021 - embopress.org
Nucleic acid‐based therapeutics that regulate gene expression have been developed
towards clinical use at a steady pace for several decades, but in recent years the field has …

Recent progress in non-native nucleic acid modifications

LK McKenzie, R El-Khoury, JD Thorpe… - Chemical Society …, 2021 - pubs.rsc.org
While Nature harnesses RNA and DNA to store, read and write genetic information, the
inherent programmability, synthetic accessibility and wide functionality of these nucleic acids …

Organelle-level precision with next-generation targeting technologies

A Saminathan, M Zajac, P Anees… - Nature Reviews Materials, 2022 - nature.com
Intracellular organelles are subsystems within a cell, whose activity and chemical
composition reflect the metabolic state of live cells. Alterations in cellular homeostasis …

Strategies, design, and chemistry in siRNA delivery systems

Y Dong, DJ Siegwart, DG Anderson - Advanced drug delivery reviews, 2019 - Elsevier
Emerging therapeutics that utilize RNA interference (RNAi) have the potential to treat broad
classes of diseases due to their ability to reversibly silence target genes. In August 2018, the …

The chemical evolution of oligonucleotide therapies of clinical utility

A Khvorova, JK Watts - Nature biotechnology, 2017 - nature.com
After nearly 40 years of development, oligonucleotide therapeutics are nearing meaningful
clinical productivity. One of the key advantages of oligonucleotide drugs is that their delivery …

Chemistries for DNA nanotechnology

M Madsen, KV Gothelf - Chemical reviews, 2019 - ACS Publications
The predictable nature of DNA interactions enables the programmable assembly of highly
advanced 2D and 3D DNA structures of nanoscale dimensions. The access to ever larger …