Results 181 to 190 of about 72,117 (255)

Structural Characterization of <i>DDX23</i> 5' UTR Regulatory Elements and Their Targeting by LNA-Modified Antisense Oligonucleotides. [PDF]

Int J Mol Sci
Kamzeeva P, Shepelev N, Zabbarova V, Brylev V, Chistov A, Ryazantsev D, Kot E, Novopashina D, Rubtsova M, Aralov A.   +9 more
europepmc   +1 more source

Decoding Undesirable Inflammatory Responses of Nucleic Acid‐Delivering Lipid Nanoparticles

Advanced Science, Volume 13, Issue 33, 15 June 2026.
Lipid nanoparticles (LNPs) enable efficient nucleic acid delivery, but their immunogenicity is a double‐edged sword. This review explores LNP‐driven innate and adaptive immunity, covering lipid components, endosomal escape, and nucleic acid sensing.
Ruimin Hu, Yin Dou, Chenwen Li, Yuying Mu, Huiying Ouyang, Wenhao Shen, Jianxiang Zhang   +6 more
wiley   +1 more source

Tofersen and other antisense oligonucleotides in ALS. [PDF]

Ther Adv Neurol Disord
Ludolph A, Wiesenfarth M.
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

Behavioral improvement in dystrophic <i>mdx23</i> mouse following repeated antisense oligonucleotides injections. [PDF]

Mol Ther Nucleic Acids
Aghaeipour A, Mitsogiannis M, Fergus C, Tetorou K, Gileadi T, Stojek E, Talavera S, Singh S, Siddle M, Prendeville J, Sokolowska E, Kelly V, Morgan J, Montanaro F, Muntoni F.   +14 more
europepmc   +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

Expansion of Splice-Switching Therapy with Antisense Oligonucleotides. [PDF]

Int J Mol Sci
Takeshima Y.
europepmc   +1 more source

The hsa_circ_0070354/miR‐4322/RAB3B Axis Facilitates the Carcinogenesis and Development of Non‐Small Cell Lung Cancer (NSCLC)

Cancer Medicine, Volume 15, Issue 6, June 2026.
ABSTRACT Background Circular RNA (hsa_circ_0070354), also known as circPTPN13, was demonstrated in our previous study that it can be used as a biomarker for diagnosing non‐small cell lung cancer (NSCLC). However, the mechanism of its role in the occurrence and development of NSCLC has not been investigated.
Yuejiao Huang, Shiyi Qin, Yu Zhang, Xinliang Gu, Ming Zheng, Guojie Ling, Lei Yang, Shaoqing Ju   +7 more
wiley   +1 more source

SNA-modified antisense oligonucleotides: A new pathway for renal targeting? [PDF]

Mol Ther Nucleic Acids
Dumoulin B, Yamada K, Susztak K.
europepmc   +1 more source