Results 121 to 130 of about 127,002 (321)

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

Tissue pharmacokinetics of antisense oligonucleotides

Molecular Therapy: Nucleic Acids
Pharmacokinetics (PK) of antisense oligonucleotides (ASOs) is characterized by rapid distribution from plasma to tissue and slow terminal plasma elimination driven by re-distribution from tissue.
Erica Bäckström, Alessandro Bonetti, Per Johnsson, Stefan Öhlin, Anders Dahlén, Patrik Andersson, Shalini Andersson, Peter Gennemark   +7 more
doaj   +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

mRNA fusion constructs serve in a general cell‐based assay to profile oligonucleotide activity [PDF]

, 2017
A cellular assay has been developed to allow measurement of the inhibitory activity of large numbers of oligonucleotides at the protein level. The assay is centred on an mRNA fusion transcript construct comprising of a full‐length reporter gene with a ...
Asselbergs, Fred, Hall, Jonathan, Häner, Robert, Hüsken, Dieter, Kinzel, Bernd, Martin, Pierre, Natt, Francois, Weiler, Jan   +7 more
core  

Vacancy defect‐induced electron homing breaks phosphodiester bonds for RNA depletion‐driven cancer therapy

BMEMat, EarlyView.
Illustration of 5% S‐vacancy Bi2S3 mediated phosphodiester bonds cleavage in RNA of hepatocellular carcinoma cells, which suppressing ERI3 expression, inhibiting cell proliferation and promoting apoptosis. Abstract Genome‐wide hypertranscription is a hallmark of malignant progression.
Chuncheng Yang, Zehao Shen, Guangming Xiang, Zhe Liu, Huaxin Liu, Jiabei Li, Ao Wang, Yelin Wu, Peiran Zhao, Xingwu Jiang, Jinghan Wang, Xiaoqing Jiang, Wenbo Bu   +12 more
wiley   +1 more source

Distribution and stability of antisense phosphorothioate oligonucleotides in rodent brain following direct intraparenchymal controlled-rate infusion [PDF]

, 1997
William C. Broaddus, Sujit S. Prabhu, G. T. Gillies, Jeffrey Neal, William S. Conrad, Zhijian Chen, Helen L. Fillmore, Harold F. Young   +7 more
openalex   +1 more source

Recent Insights Into Alzheimer's Disease: Advances in Pathophysiology and Therapeutic Strategies

Brain Health, EarlyView.
ABSTRACT Alzheimer's disease (AD) is a complex neurodegenerative disorder driven by interplay among amyloid‐β (Aβ) plaques, tau neurofibrillary tangles, inflammation and comorbid pathologies. Recent advances in biomarkers and therapeutic approvals have reshaped our understanding of its pathophysiology and treatment landscape.
Jiong Shi, Jeffery Cummings, Jacques Touchon, Bruno Vellas   +3 more
wiley   +1 more source

Evolved DNAzymes and Stable Activation Chemistry Enable High‐Efficiency DNA Ligation

Chemistry – A European Journal, EarlyView.
In vitro selection of pre‐structured DNA libraries and chemical screening of alternative activation groups yielded trans‐acting DNAzymes with faster ligation rates and enhanced DNA substrate stability. The combination of C3S1 DNAzymes and phosphobenzimidazole‐activated DNA substrates provides improved DNA ligation performance, offering promise for ...
Connor Nurmi, Gemma Mendonsa, Mengdi Bao, Yingfu Li   +3 more
wiley   +1 more source

Investigation of the activity of phosphothioate and phosphothioate-LNA-modified oligonucleotides against HIV-1

Frontiers in Medicine
This study investigated the antiretroviral efficacy, toxicity profile, and cellular uptake of phosphothioate (PS) and PS/LNA-modified oligonucleotides within an in vitro HIV infection model.
Ludmila Gotfrid, Kirill Elfimov, Maria Gashnikova, Aleksey Totmenin, Aleksandr Agaphonov, Natalya Gashnikova   +5 more
doaj   +1 more source

Molecular analysis and phenotype characterization of the progeny of two antisense potato plants [PDF]

, 2008
Two transgenic potato lines csr2-1 and csr4-8, containing two different antisense constructs, csr2 and csr4, respectively, were crossed to investigate the possibility of achieving double transformants with combined effects of the two antisense transgenes
Jacobsen, E., Olawole, O., Visser, R.G.F.   +2 more
core   +1 more source