Results 251 to 260 of about 1,703,096 (348)

Antisense Oligonucleotide Therapy for Amyotrophic Lateral Sclerosis (ALS): An Umbrella Review. [PDF]

Cureus
Jeong E, Li D.
europepmc   +1 more source

A Phase 1, Randomized, Open‐Label, Parallel Group Study to Evaluate the Relative Bioavailability and Safety of Subcutaneous Bepirovirsen when Delivered from a Vial or Prefilled Syringe Fitted with a Safety Syringe Device in Healthy Adult Participants

Clinical Pharmacology in Drug Development, Volume 15, Issue 2, February 2026.
Abstract Bepirovirsen, an antisense oligonucleotide in development for the treatment of chronic hepatitis B virus (HBV) infection, is administered from glass vials as a subcutaneous (SC) injection by healthcare professionals (HCPs). A ready‐to‐use prefilled syringe (PFS) assembled with a safety syringe device (SSD) has been developed to make ...
Amir S. Youssef, Poonam Shah, Maxwell Hu, Helene Plein, Abhishek Roy, Ravi Sharma, Sarah Mole, Magdalena Blazejczyk, Wendy Cross, Brian Spears, Samuel Pak, Rejbinder Kaur, Robert Elston, Dickens Theodore, Marjan Hezareh, Ahmed Nader   +15 more
wiley   +1 more source

Exploitation of Biocomputational Approaches for siRNA-Mediated Gene Silencing of HPV: A Novel Therapeutic Strategy for Cervical Cancer. [PDF]

Cancer Rep (Hoboken)
Masum MHU, Parvin R, Heema HP, Akter A, Ferdous J.   +4 more
europepmc   +1 more source

Transient Cyclic Structured Oligonucleotide Designs for Therapeutic Applications

Current Protocols, Volume 6, Issue 2, February 2026.
Abstract Oligonucleotide‐based therapeutics are now widely used in clinical settings. From the late 1980s to the mid‐1990s, efforts to improve therapeutic efficacy focused on imparting drug‐like properties to oligonucleotides, emphasizing nuclease stability and target sequence affinity.
Sudhir Agrawal
wiley   +1 more source

Dystrophin rescue in the brain for DMD. [PDF]

Mol Ther Nucleic Acids
Li M, Han R.
europepmc   +1 more source

Implications for Cardiac Function Following Rescue of the Dystrophic Diaphragm in a Mouse Model of Duchenne Muscular Dystrophy [PDF]

A Asai, A Crisp, A De Troyer, A Malerba, A Malerba, A Malerba, BJ Petrof, C Betts, C Kirchmann, CF Spurney, DJ Stuckey, DM Connuck, G Dickson, G Nigro, H Yin, H Yin, J Alter, J Kuroda, J Morrison, JG Quinlan, JM Costas, JO Benditt, JW Carnwath, K Ito, K Ohlendieck, L Loufrani, L Loufrani, L Loufrani, M Ishizaki, M Ishizaki, M Mosqueira, M Palladino, MG Dunckley, N Jearawiriyapaisarn, P Melacini, QL Lu, S Fletcher, SD Wilton   +37 more
core   +1 more source

Areas of research priorities in epilepsy: A position paper of the European Reference Network for Rare and Complex Epilepsies, EpiCARE

Epilepsia Open, Volume 11, Issue 1, Page 291-321, February 2026.
Abstract Objective To define and articulate research priorities in epilepsy identified by the European Reference Network for Rare and Complex Epilepsies (ERN EpiCARE), addressing key unmet needs across the spectrum of rare and complex epilepsies. Methods This position paper was developed through a structured collaborative process involving patient ...
Sébile Tchaicha, Stéphane Auvin, Sándor Beniczky, Andreas Brunklaus, Lieven Lagae, Emilio Perucca, Rainer Surges, Sophie Adler, Christoph Helmstaedter, Floor Jansen, Guido Rubboli, Philippe Ryvlin, Nicola Specchio, Eugen Trinka, Ingmar Blümcke, Valentina de Giorgis, Katarzyna Kotulska, Gaetan Lesca, Masa Malenica, Amy McTague, Rima Nabbout, Rikke Steensbjerre Møller, Sandra Silva Arrieta, Isabella Brambilla, Małgorzata Kosla, Kees Braun, J. Helen Cross, Alexis Arzimanoglou   +27 more
wiley   +1 more source

Targeting <i>carA</i> Using Optimized Antisense Peptide Nucleic Acid-Cell-Penetrating Peptide Conjugates in <i>Acinetobacter baumannii</i>: A Novel Antibacterial Approach. [PDF]

Int J Mol Sci
Seo JH, Kim YJ, Jeon WJ, Yoo JS, Moon DC.   +4 more
europepmc   +1 more source

Astrocyte MCT1 Expression Does Not Contribute to the Axonal Degenerative Phenotype Observed With Ubiquitous MCT1 Depletion

Glia, Volume 74, Issue 2, February 2026.
Loss of astrocytic MCT1 does not cause late onset neurodegeneration. As ubiquitous MCT1 deletion causes axonal degeneration, oligodendrocytes and potentially other cells are more prominent drivers of MCT1‐mediated metabolic support of neurons. ABSTRACT We recently reported that the loss of oligodendrocyte metabolic support through the lactate and ...
Thomas Philips, Emily G. Thompson, Olivia Spead, Balaji G. Vijayakumar, Erica R. Kent, Sean J. Miller, Treasure Nwokeleme, Svetlana Vidensky, Mohamed Hassan Farah, Jeffrey D. Rothstein   +9 more
wiley   +1 more source

Ribofuranose-based GalNAc-conjugated siRNA enhances the liver-targeted delivery and elicits robust RNAi-mediated gene silencing. [PDF]

Mol Ther Nucleic Acids
Huang ZA, Li ZM, Wang J, Ji GS, Gao ZC, Chen AF, Tian ZK, Yu F, Song GS.   +8 more
europepmc   +1 more source