Results 221 to 230 of about 131,722 (303)

<i>In vivo</i> delivery of a fluorogenic antisense oligonucleotide into human leukocytes in a humanized mouse model system. [PDF]

Mol Ther Methods Clin Dev
Seidel NB, Rout SS, Packard BZ, Lavender KJ.   +3 more
europepmc   +1 more source

Novel drugs approved by the EMA, the FDA and the MHRA in 2025: A year in review

British Journal of Pharmacology, Volume 183, Issue 9, Page 1779-1813, May 2026.
Abstract In the 2025 novel drug mini‐review, one can take a full measure of the ingenuity that underlies current drug design and development, despite the year's smaller harvest (46 novel drugs) compared to 2024 (53) and 2023 (70). 54% of the novel drugs are first‐in‐class (FIC).
Andreas Papapetropoulos, Stavros Topouzis, Steve P. H. Alexander, Miriam M. Cortese‐Krott, Zsuzsanna Helyes, Kirill Martemyanov, Claudio Mauro, Nithyanandan Nagercoil, Reynold A. Panettieri Jr, Hemal H. Patel, Rainer Schulz, Barbara Stefanska, Gary J. Stephens, Nathalie Vergnolle, Xin Wang, Stephen Ward, Péter Ferdinandy   +16 more
wiley   +1 more source

A splice-switching antisense oligonucleotide approach for pediatric genetic epilepsies

Dame HB, Kopalle H, Miñana B, Klaft Z, Fahey JB, McWatters DC, Rao V, Suresh R, Aigner S, Hammer MF, Yuskaitis CJ, Wong M, Teaney N, Zhang C, Thompson M, Lehner B, Bennett CF, Valcárcel J, Yeo GW, Burge CB, Dulla CG, Oudin MJ.   +21 more
europepmc   +1 more source

Therapeutic antisense oligonucleotide mitigates retinal dysfunction in a pig model of CLN3 Batten disease. [PDF]

Nucleic Acids Res
Stratton MP, Centa JL, Swier VJ, Pfeifer WL, Booth CD, Albert K, Hunyara JL, Rechtzigel MJ, Duelli FJ, Leppert HG, Rigo F, Smit T, Jafar-Nejad P, Weimer JM, Drack AV, Hastings ML.   +15 more
europepmc   +1 more source

Rebalancing the Neurovascular Axis: NTRK2 Isoform Equilibrium as a Determinant of Pulmonary Endothelial Regeneration

Cell Proliferation, Volume 59, Issue 5, May 2026.
Summary of the functions of the NTRK2 receptor during lung injury and regeneration. Our recent work using both human organoid and mouse disease models highlights the non‐conventional role of NTRK2 isoforms in regulating pulmonary vasculature and alveolar regeneration during bronchopulmonary dysplasia.
Gan Zhou, Cheng Tan, Yifei Miao
wiley   +1 more source

Targeting Bacterial Adenylate Kinase mRNA with a Chimeric Antisense Oligonucleotide for Rational Antibacterial Drug Development. [PDF]

Molecules
Otcheva LA, Traykovska M, Penchovsky R.
europepmc   +1 more source

Optimizing Oligonucleotide Therapeutics: A Model‐Informed Drug Development Perspective

Clinical and Translational Science, Volume 19, Issue 5, May 2026.
ABSTRACT Oligonucleotide therapies have emerged as a powerful therapeutic class, providing novel solutions for diverse diseases through antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), and aptamers that specifically target and modulate gene expression or protein function. However, oligonucleotide development faces distinct challenges,
Ye Yuan, Vishnu Sharma, Venkatesh Atul Bhattaram, Xiaolei Pan, Justin Earp, Yun Wang, Jiang Liu, Hao Zhu   +7 more
wiley   +1 more source

Peripherally administered androgen receptor-targeted antisense oligonucleotide rescues spinal pathology in a murine SBMA model. [PDF]

J Clin Invest
Lee C, Yu Z, Kuo CJ, Tejwani L, Grijalva RM, Bae E, Zhao HT, Lim J, Lieberman AP.   +8 more
europepmc   +1 more source

SORBS2: A Molecular Nexus in Multisystem Diseases Through Scaffold‐Mediated Regulation

Journal of Cellular and Molecular Medicine, Volume 30, Issue 9, May 2026.
ABSTRACT Sorbin and SH3 Domain Containing 2 (SORBS2), a multifunctional scaffold protein harbouring Sorbin homology (SoHo) and Src homology 3 (SH3) domains, serves as a molecular hub in human diseases by integrating cytoskeletal remodelling, signal transduction and RNA metabolic regulation. This study systematically analyses SORBS2's molecular features,
Qiwei Jia, Yong Zhang
wiley   +1 more source