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Delivery of oligonucleotide therapeutics: chemical modifications, lipid nanoparticles, and extracellular vesicles
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) …
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
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 …
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
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 …
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 …
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 …
disorder. FECD impacts 4% of US population and is the leading indication for corneal …
Muscle atrophy: present and future
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 …
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
Long non-coding RNAs (lncRNAs) are key regulators of numerous intracellular processes
leading to tumorigenesis. They are frequently deregulated in cancer, functioning as …
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 …
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 …
propensities of a series of fatty acid-modified locked nucleic acid (LNA) antisense …
Towards the enzymatic synthesis of phosphorothioate containing LNA oligonucleotides
Therapeutic oligonucleotides require the addition of multiple chemical modifications to the
nucleosidic scaffold in order to improve their drug delivery efficiency, cell penetration …
nucleosidic scaffold in order to improve their drug delivery efficiency, cell penetration …