Results 151 to 160 of about 45,133 (250)

Blood SOD1 Activity in ALS Patients Receiving Tofersen Treatment

Annals of Neurology, EarlyView.
Objective The antisense oligonucleotide tofersen is the first disease‐modifying drug for SOD1‐related amyotrophic lateral sclerosis (ALS) and was approved because of its ability to reduce SOD1 protein and neurofilament levels. The effect of tofersen on SOD1 activity is unclear but of clinical relevance because homozygous SOD1 mutations, linked to ...
Katharina Goehring, Enya Abler, Jakub Vavra, Kristina Mayer, Johannes Dorst, Zeynep Elmas, Maximilian Wiesenfarth, Lars Richter, Susanne Petri, Martin Regensburger, Peter Reilich, Bogdan Bjelica, Rea Lumi, Patrick Süß, Hayrettin Tumani, Markus Otto, Jochen H. Weishaupt, Albert C. Ludolph, Patrick Oeckl   +18 more
wiley   +1 more source

Multimodal Characterization of Glymphatic‐Related Magnetic Resonance Imaging Markers in Huntington's Disease: A Multi‐Cohort Retrospective Study

Annals of Neurology, EarlyView.
Objective To characterize magnetic resonance imaging (MRI)‐based glymphatic surrogates in Huntington's disease (HD) using MRI measures of perivascular diffusivity and structural perivascular alterations across multiple large cohorts. Methods We analyzed 2,731 MRI sessions from 880 participants across 3 large retrospective HD cohorts.
Alexia Solomon, Zexi Wang, Francesco Sanvito, Zheng Zhou, Adys Mendizabal, Jingwen Yao   +5 more
wiley   +1 more source

Natural antisense with human cancer

, 2009
[[abstract]]Natural antisense transcripts (NATs) were first described in prokaryotes, and also were found in the eukaryotic cell later. The ‘antisense’ term includes transcripts containing long sequences complementary to sense transcripts, and the ...
Jau-Jie You
core  

Ursolic Acid Alleviates Orchitis via Modulating Ferroptosis in Peritubular Myoid Cells of Testis

Animal Research and One Health, EarlyView.
Ursolic acid modulates key ferroptosis‐related pathways in the peritubular myoid cells of the testis in forest musk deer. ABSTRACT Chronic stress adversely affects reproductive health, increasing susceptibility to pathologies such as orchitis. Additionally, the maintenance of iron homeostasis is essential for preserving the function of the testicles ...
Yang Wang, Tianqi Jin, Zexin Chen, Ye Gong, Qimin Liu, Zifang Wu, Heran Cao, Yan Li, Yuyan Liu, Tianhao Yang, Zhihong Jiang, Wuzi Dong   +11 more
wiley   +1 more source

Switching disease‐modifying therapies in patients with spinal muscular atrophy: A systematic review on effectiveness outcomes

British Journal of Clinical Pharmacology, EarlyView.
With multiple disease‐modifying therapies now available, treatment switching has become an important clinical consideration in the management of spinal muscular atrophy (SMA). While some switches are prompted by suboptimal clinical response, more commonly they are driven by treatment burden, convenience, or adverse events.
Andrej Belančić, Elvira Meni Maria Gkrinia, Patrick Eustaquio, Andrea Katrin Faour, Dinko Vitezić   +4 more
wiley   +1 more source

Cost‐utility analysis of nusinersen–risdiplam switch in patients with spinal muscular atrophy in Croatia: A discrete event simulation model

British Journal of Clinical Pharmacology, EarlyView.
Introduction In recent years, the treatment of spinal muscular atrophy (SMA), a rare disease, has significantly progressed, improving patients' survival and overall quality of life. However, current SMA treatments are expensive, and some (nusinersen) are very inconvenient for patients.
Andrej Belančić, Ivana Stević, Elvira Meni Maria Gkrinia, Dinko Vitezić, Slobodan Janković   +4 more
wiley   +1 more source

Precision medicine in paediatrics: Progress and priorities

British Journal of Clinical Pharmacology, EarlyView.
Precision medicine is revolutionizing personalized healthcare, advancing both diagnostics and therapeutics at an unprecedented pace. Reviewing the paediatric applications of pharmacometrics, pharmacogenomics and advanced therapy medicinal products highlights not only the relevance of these exciting innovations to frontline care but also the significant
Nicola Husain, Claire Booth, Joseph F. Standing, Charlotte I. S. Barker   +3 more
wiley   +1 more source

Decoding RNA regulation: Challenges and opportunities for RNA‐based therapies in Europe

British Journal of Clinical Pharmacology, EarlyView.
Abstract RNA‐based medicinal products represent a promising frontier in personalised medicine, offering sequence‐specific disease targeting at various molecular levels, yet their clinical translation in the European Union (EU) may be hindered by regulatory uncertainty around definitions and evidence requirements; this study therefore aims to identify ...
Olivia C. Lewis, Christine Leopold, Joyce M. Hoek, Raymond M. Schiffelers, Marie L. De Bruin   +4 more
wiley   +1 more source

4′‐ThioRNA Substitution and Sense‐Strand Segmentation for the Design of Functional Fully Modified siRNAs Targeting SARS‐CoV‐2

Chemistry – A European Journal, EarlyView.
4′‐ThioRNA was broadly tolerated across multiple positions in SARS‐CoV‐2‐targeting siRNAs, but full 4'‐thio modification of both strands caused a reduction in RNAi activity. Sense‐strand segmentation improved RNAi and antiviral activity, particularly when the nick was located at position 10 from the 5′‐end of the sense strand, thereby unlocking the ...
Yuhei Nogi, Jun Tsukimoto, Noriko Saito‐Tarashima, Takaaki Koma, Masako Nomaguchi, Noriaki Minakawa   +5 more
wiley   +1 more source

Multivalent RNA‐Cleaving Agents on a Cubic Octameric Silsesquioxane Core

Chemistry – A European Journal, EarlyView.
RNA‐cleaving agents on a cubic octameric silsesquioxane core were synthesized and characterized. Several catalytic systems of these structures were screened using hexaribonucleotide models. The best systems were then further used to study their catalytic efficiency in cleaving a HER2 mRNA model sequence. The induced cleavage sites were also determined.
Hanni Haapsaari, Iris Tuomi, Pasi Virta
wiley   +1 more source