Delivery of oligonucleotide therapeutics: chemical modifications, lipid nanoparticles, and extracellular vesicles

JP Bost, H Barriga, MN Holme, A Gallud, M Maugeri… - ACS …, 2021 - ACS Publications
Oligonucleotides (ONs) comprise a rapidly growing class of therapeutics. In recent years, the
list of FDA-approved ON therapies has rapidly expanded. ONs are small (15–30 bp) …

Enhancing the effectiveness of oligonucleotide therapeutics using cell-penetrating peptide conjugation, chemical modification, and carrier-based delivery strategies

S Anwar, F Mir, T Yokota - Pharmaceutics, 2023 - mdpi.com
Oligonucleotide-based therapies are a promising approach for treating a wide range of hard-
to-treat diseases, particularly genetic and rare diseases. These therapies involve the use of …

Intratracheally administered LNA gapmer antisense oligonucleotides induce robust gene silencing in mouse lung fibroblasts

M Shin, IL Chan, Y Cao, AM Gruntman… - Nucleic Acids …, 2022 - academic.oup.com
The lung is a complex organ with various cell types having distinct roles. Antisense
oligonucleotides (ASOs) have been studied in the lung, but it has been challenging to …

The lncRNA punisher regulates apoptosis and mitochondrial homeostasis of vascular smooth muscle cells via targeting miR‐664a‐5p and OPA1

Y Yang, M Li, Y Liu, Z Wang, X Fu, X He… - Oxidative Medicine …, 2022 - Wiley Online Library
Long noncoding RNAs (lncRNAs) are important regulators of various cellular functions.
Recent studies have shown that a novel lncRNA termed Punisher is highly expressed in …

Oligonucleotides targeting TCF4 triplet repeat expansion inhibit RNA foci and mis-splicing in Fuchs' dystrophy

J Hu, Z Rong, X Gong, Z Zhou… - Human molecular …, 2018 - academic.oup.com
Fuchs' endothelial corneal dystrophy (FECD) is the most common repeat expansion
disorder. FECD impacts 4% of US population and is the leading indication for corneal …

Muscle atrophy: present and future

RY Cao, J Li, Q Dai, Q Li, J Yang - Muscle atrophy, 2018 - Springer
Muscle atrophy is the loss of muscle mass and strength, and it occurs in many diseases,
such as cancer, AIDS (acquired immunodeficiency syndrome), congestive heart failure …

[HTML][HTML] Regulation of LncRNAs in melanoma and their functional roles in the metastatic process

M Melixetian, PG Pelicci, L Lanfrancone - Cells, 2022 - mdpi.com
Long non-coding RNAs (lncRNAs) are key regulators of numerous intracellular processes
leading to tumorigenesis. They are frequently deregulated in cancer, functioning as …

ADAM33′ s Role in Asthma Pathogenesis: An Overview

J Sleziak, A Gawor, M Błażejewska, K Antosz… - International Journal of …, 2024 - mdpi.com
Asthma is a complex chronic respiratory disease characterized by airway
hyperresponsiveness, inflammation, and obstruction. Many genes have been identified as …

Propensities of Fatty Acid-Modified ASOs: Self-Assembly vs Albumin Binding

EA Kusznir, JC Hau, M Portmann… - Bioconjugate …, 2023 - ACS Publications
We conducted a biophysical study to investigate the self-assembling and albumin-binding
propensities of a series of fatty acid-modified locked nucleic acid (LNA) antisense …

Towards the enzymatic synthesis of phosphorothioate containing LNA oligonucleotides

M Flamme, S Hanlon, H Iding, K Puentener… - Bioorganic & Medicinal …, 2021 - Elsevier
Therapeutic oligonucleotides require the addition of multiple chemical modifications to the
nucleosidic scaffold in order to improve their drug delivery efficiency, cell penetration …