Results 161 to 170 of about 72,117 (254)

MicroRNAs in cancer: biogenesis, biomarkers and therapeutic strategies. [PDF]

Front Med (Lausanne)
Rawat N, Babu N, Hussain A, Panwar AS.
europepmc   +1 more source

Digital twins to accelerate target identification and drug development for immune‐mediated disorders

FEBS Open Bio, Volume 16, Issue 6, Page 1048-1059, June 2026.
Digital twins integrate patient‐derived molecular and clinical data into personalised computational models that simulate disease mechanisms. They enable rapid identification and validation of therapeutic targets, prediction of drug responses, and prioritisation of candidate interventions.
Anna Niarakis, Philippe Moingeon
wiley   +1 more source

Dynamics of an RNase H-Responsive Tetrahedral DNA Nanostructure for Efficient Intracellular microRNA Inhibition. [PDF]

Bioconjug Chem
Martins ASG, Reis SD, Baboi R, Furlan F, Cortinhas J, Carvalho FA, Santos NC, Bath J, Benson E, Pêgo AP, Moreno PMD.   +10 more
europepmc   +1 more source

Characterization of Clinical Phenotype to Glial Fibrillary Acidic Protein Concentrations in Alexander Disease

Annals of Clinical and Translational Neurology, Volume 13, Issue 6, Page 1206-1221, June 2026.
ABSTRACT Objective To determine the concentration of glial fibrillary acidic protein (GFAP) in cerebrospinal fluid (CSF) and plasma in Alexander disease (AxD) and whether GFAP levels are predictive of disease phenotypes. Methods CSF and plasma were collected (longitudinally when available) from AxD participants and non‐AxD controls.
Amy T. Waldman, Asako Takanohashi, Joshua Y. Joung, Geraldine W. Liu, Kaley Arnold, Amy Pizzino, Walter Faig, Sarah Woidill, Sona Narula, Adeline L. Vanderver   +9 more
wiley   +1 more source

Application of Antisense Oligonucleotides as an Alternative Approach for Gene Expression Control and Functional Studies. [PDF]

Int J Mol Sci
Szukowska A, Żuk M, Sztompke J, Bednarz B, Kaźmierczak U.   +4 more
europepmc   +1 more source

First Generation Proteolysis Targeting Chimeras (PROTACs) for the Treatment of Progeria

Advanced Science, Volume 13, Issue 32, 9 June 2026.
We report the first PROTACs designed to degrade progerin, introducing a novel therapeutic approach for progeria. The best compound, UCM‐18142, significantly reduces progerin levels and improves key disease phenotypes in patient‐derived cells and in the LmnaG609G/G609G mouse model, paving the way for new treatment strategies targeting the root cause of ...
Jon Macicior‐Michelena, Marcelino Telechea, Daniel Fernández, Alba García‐Martín, Ángeles Canales, Silvia Ortega‐Gutiérrez   +5 more
wiley   +1 more source

Silencing HPV: the rise of RNA therapeutics in cervical cancer. [PDF]

Infect Agent Cancer
Khorramabadi SM, Ebrahimi N, Aghbash PS, Ravanlo ZZ, Baghi HB.   +4 more
europepmc   +1 more source

High Prevalence of SOD1 Pathogenic Variants in the UK Biobank: Implications for Early Intervention in Amyotrophic Lateral Sclerosis

Annals of Neurology, Volume 99, Issue 6, Page 1502-1515, June 2026.
Objective SOD1 is the second most frequently mutated gene in European patients with amyotrophic lateral sclerosis (ALS). Given the recent authorization of SOD1‐targeted antisense oligonucleotides for SOD1‐ALS, prompt screening for SOD1 mutations in patients with ALS patients is highly recommended.
Delia Gagliardi, Chiara Villella, Matteo Zanovello, Virginia Iacobelli, Stefania Corti, Giacomo Pietro Comi, Pietro Fratta, Henry Houlden, Arianna Tucci, Dario Ronchi   +9 more
wiley   +1 more source

The evolving therapeutic landscape of spinal muscular atrophy – A scoping review of investigational agents, emerging delivery technologies and strategic innovations

British Journal of Clinical Pharmacology, Volume 92, Issue 6, Page 1552-1566, June 2026.
Spinal muscular atrophy (SMA) is a severe neuromuscular disease with emerging therapeutic complexity. This review aims to systematically map the global pipeline of investigational treatments for SMA. Using ClinicalTrials.gov and complementary international registries, we identified 21 planned or ongoing interventional trials from 2020 to 2025 targeting
Andrej Belančić, Patrick Eustaquio, Elvira Meni Maria Gkrinia, Ivana Stević, Eugen Javor, Yun Wah Lam, Slobodan Janković, Dinko Vitezić   +7 more
wiley   +1 more source

Effects and mechanisms of mascRNA regulate the proliferation, migration and invasion of Laryngeal Squamous Cell Carcinoma in vitro. [PDF]

Braz J Otorhinolaryngol
Peng RJ, Zhang Z, Zhu S, Wu Y, Xu A, Li H, Ma S, Wang X.   +7 more
europepmc   +1 more source