Results 31 to 40 of about 549 (211)

Modified ASO conjugates encapsulated with cytidinyl/cationic lipids exhibit more potent and longer-lasting anti-HCC effects

Molecular Therapy: Nucleic Acids, 2023
Antisense oligonucleotides (ASOs) are a class of therapeutics targeting mRNAs or genes that have attracted much attention. However, effective delivery and optimal accumulation in target tissues in vivo are still challenging issues.
Yufei Pan, Jing Guan, Yujing Gao, Yuejie Zhu, Huantong Li, Hua Guo, Qianyi He, Zhu Guan, Zhenjun Yang   +8 more
doaj   +1 more source

Identifying transcription factors controlling the basal expression of human MRP4 highlights a substantial role for Sp1

FEBS Open Bio, EarlyView.
The MRP4 transporter exports several drugs and signaling molecules. Here, we identified key promoter elements regulating basal MRP4 expression. Using reporter assays, we defined a conserved region with essential Sp1 and contributory Ets sites, which controlled basal MRP4 expression.
Debora Singer, Angelique Kragl, Katrin Ziems, Juliane Glaubitz, Sophie Grammbauer, Susanne Hildebrandt, Mladen V. Tzvetkov, Gabriele Jedlitschky   +7 more
wiley   +1 more source

Efficient functional neutralization of lethal peptide toxins in vivo by oligonucleotides

Scientific Reports, 2017
Medical means to save the life of human patients affected by drug abuse, envenomation or critical poisoning are currently limited. While the compounds at risks are most often well identified, particularly for bioterrorism, chemical intervention to ...
Tarek Mohamed Abd El-Aziz, Corinne Ravelet, Jordi Molgo, Emmanuelle Fiore, Simon Pale, Muriel Amar, Sawsan Al-Khoury, Jérôme Dejeu, Mahmoud Fadl, Michel Ronjat, Germain Sotoing Taiwe, Denis Servent, Eric Peyrin, Michel De Waard   +13 more
doaj   +1 more source

UiO‐66 metal–organic frameworks in biomedicine: From structural tunability to bioimaging, photodiagnostics, and photodynamic cancer therapy

FEBS Open Bio, EarlyView.
UiO‐66(Zr) metal–organic frameworks are chemically stable, biocompatible, and highly tunable nanomaterials. Their modular structure enables controlled drug delivery, multimodal bioimaging, and light‐activated photodynamic therapy, supporting integrated diagnostic and therapeutic (theranostic) applications in cancer and biomedical research.
Veronika Huntošová, Grigorii Rakhalskii, Miroslav Almáši   +2 more
wiley   +1 more source

Position-Dependent Stabilization of DNA/RNA Duplexes by Site-Specific Incorporation of LNA Nucleosides

Journal of Nucleic Acids
Owing to their enhanced functional properties, 2′,4′-bridged nucleic acids/locked nucleic acids (2′,4′-BNAs/LNAs) are considered promising candidates for antisense oligonucleotide therapeutics.
Elisa Tomita-Sudo, Tomoka Akita, Nae Sakimoto, Saori Tahara-Takamine, Renshin Sano, Shigenori Iwai, Junji Kawakami   +6 more
doaj   +1 more source

Guanine Modification of Inhibitory Oligonucleotides Potentiates Their Suppressive Function [PDF]

The Journal of Immunology, 2013
Abstract Inhibitory TLR7 and/or TLR9 oligonucleotides (inhibitory oligonucleotide [INH-ODN]) are characterized by a phosphorothioate backbone and a CC(T)XXX3–5GGG motif, respectively. INH-ODN 2088 is a prototypic member of this class of INH-ODN and acts as a TLR7 and TLR9 antagonist.
Franziska, Römmler, Marion, Jurk, Eugen, Uhlmann, Monika, Hammel, Anna, Waldhuber, Lavinia, Pfeiffer, Hermann, Wagner, Jörg, Vollmer, Thomas, Miethke   +8 more
openaire   +2 more sources

Remote Assessment of Ataxia Severity in SCA3 Across Multiple Centers and Time Points

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Spinocerebellar ataxia type 3 (SCA3) is a genetically defined ataxia. The Scale for Assessment and Rating of Ataxia (SARA) is a clinician‐reported outcome that measures ataxia severity at a single time point. In its standard application, SARA fails to capture short‐term fluctuations, limiting its sensitivity in trials.
Marcus Grobe‐Einsler, Vivian Maas, Arian Taheri Amin, Jennifer Faber, Tamara Schaprian, Katharina Hill, Matthis Synofzik, Lisa H. Graf, Heike Jacobi, Manuela Lima, Ana F. Ferreira, Bart P. van de Warrenburg, Ilse H. J. Willemse, Dagmar Timmann, Andreas Thieme, Paola Giunti, Hector Garcia‐Moreno, Magda M. Santana, Jeannette Hübener‐Schmid, Elin H. Davies, Thomas Klockgether   +20 more
wiley   +1 more source

Gene Expression Profile Changes After Short-activating RNA-mediated Induction of Endogenous Pluripotency Factors in Human Mesenchymal Stem Cells

Molecular Therapy: Nucleic Acids, 2012
It is now recognized that small noncoding RNA sequences have the ability to mediate transcriptional activation of specific target genes in human cells.
Jon Voutila, Pål Sætrom, Paul Mintz, Guihua Sun, Jessica Alluin, John J Rossi, Nagy A Habib, Noriyuki Kasahara   +7 more
doaj   +1 more source

Cell-Penetrating Peptides and Transportan

Pharmaceutics, 2021
In the most recent 25–30 years, multiple novel mechanisms and applications of cell-penetrating peptides (CPP) have been demonstrated, leading to novel drug delivery systems.
Ülo Langel
doaj   +1 more source

Functional and Structural Evidence of Neurofluid Circuit Aberrations in Huntington Disease

Annals of Clinical and Translational Neurology, EarlyView.
ABSTRACT Objective Disrupted neurofluid regulation may contribute to neurodegeneration in Huntington disease (HD). Because neurofluid pathways influence waste clearance, inflammation, and the distribution of central nervous system (CNS)–delivered therapeutics, understanding their dysfunction is increasingly important as targeted treatments emerge.
Kilian Hett, Abigail Dubois, Melanie Leguizamon, Alexander Song, Paula Trujillo, Colin D. McKnight, Ciaran M. Considine, Manus J. Donahue, Daniel O. Claassen   +8 more
wiley   +1 more source