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 …

RNA modifications in physiology and disease: towards clinical applications

S Delaunay, M Helm, M Frye - Nature Reviews Genetics, 2024 - nature.com
The ability of chemical modifications of single nucleotides to alter the electrostatic charge,
hydrophobic surface and base pairing of RNA molecules is exploited for the clinical use of …

[HTML][HTML] Peptide-based nanomaterials: Self-assembly, properties and applications

T Li, XM Lu, MR Zhang, K Hu, Z Li - Bioactive materials, 2022 - Elsevier
Peptide-based materials that have diverse structures and functionalities are an important
type of biomaterials. In former times, peptide-based nanomaterials with excellent stability …

siRNA: Mechanism of action, challenges, and therapeutic approaches

W Alshaer, H Zureigat, A Al Karaki, A Al-Kadash… - European journal of …, 2021 - Elsevier
Owing to specific and compelling gene silencing, RNA interference (RNAi) is expected to
become an essential approach in treating a variety of infectious, hemato-oncological …

Polymeric delivery of therapeutic nucleic acids

R Kumar, CF Santa Chalarca, MR Bockman… - Chemical …, 2021 - ACS Publications
The advent of genome editing has transformed the therapeutic landscape for several
debilitating diseases, and the clinical outlook for gene therapeutics has never been more …

[HTML][HTML] Cancer nanomedicine for combination cancer immunotherapy

J Nam, S Son, KS Park, W Zou, LD Shea… - Nature Reviews …, 2019 - nature.com
Cancer immunotherapy is revolutionizing oncology. However, dose-limiting toxicities and
low patient response rates remain major challenges in the clinic. Cancer nanomedicine in …

Therapeutic antisense oligonucleotides are coming of age

CF Bennett - Annual review of medicine, 2019 - annualreviews.org
The first published description of therapeutic applications of antisense oligonucleotide
(ASO) technology occurred in the late 1970s and was followed by the founding of …

Antisense oligonucleotides: an emerging area in drug discovery and development

K Dhuri, C Bechtold, E Quijano, H Pham… - Journal of clinical …, 2020 - mdpi.com
Antisense oligonucleotides (ASOs) bind sequence specifically to the target RNA and
modulate protein expression through several different mechanisms. The ASO field is an …

A tubular DNA nanodevice as a siRNA/chemo‐drug co‐delivery vehicle for combined cancer therapy

Z Wang, L Song, Q Liu, R Tian, Y Shang… - Angewandte …, 2021 - Wiley Online Library
Using the DNA origami technique, we constructed a DNA nanodevice functionalized with
small interfering RNA (siRNA) within its inner cavity and the chemotherapeutic drug …

Visualization of the process of a nanocarrier-mediated gene delivery: stabilization, endocytosis and endosomal escape of genes for intracellular spreading

Z Ma, Y Zheng, Z Chao, H Chen, Y Zhang… - Journal of …, 2022 - Springer
Nanoparticles have been widely applied as gene carrier for improving RNA interference
(RNAi) efficiency in medical and agricultural fields. However, the mechanism and delivery …