Results 61 to 70 of about 124,170 (327)
Annals of Clinical and Translational Neurology, EarlyView.ABSTRACT Objective Alexander disease (AxD) is a severe neurodegenerative disorder caused by gain‐of‐function mutations in the gene for GFAP, which lead to protein aggregation and a primary astrocytopathy. Symptoms vary, but failure to thrive (FTT) and frequent emesis are common and cause significant morbidity. Here we investigate GDF15, a member of the
Tracy L. Hagemann +6 more
wiley +1 more source
Molecular Mechanisms of Antisense Oligonucleotides [PDF]
Nucleic Acid Therapeutics, 2017 In 1987, when I became interested in the notion of antisense technology, I returned to my roots in RNA biochemistry and began work to understand how oligonucleotides behave in biological systems. Since 1989, my research has focused primarily on this topic, although I have been involved in most areas of research in antisense technology.
openaire +3 more sources
Antisense oligonucleotides for the treatment of dyslipidaemia [PDF]
European Heart Journal, 2012 Antisense oligonucleotides (ASOs) are short synthetic analogues of natural nucleic acids designed to specifically bind to a target messenger RNA (mRNA) by Watson-Crick hybridization, inducing selective degradation of the mRNA or prohibiting translation of the selected mRNA into protein.
Joseph L. Witztum +3 more
openaire +3 more sources
Advanced Functional Materials, EarlyView.Many known diseases arise from dysregulated gene expression, and differentially expressed genes can serve as biomarkers to distinguish diseased cells from healthy tissues. In this study, reconfigurable nucleic acid nanoparticles (recNANPs) are introduced that can detect overexpressed cancer biomarkers and subsequently release RNAi inducers to silence ...
Yelixza I. Avila +7 more
wiley +1 more source
The Journal of Qazvin University of Medical Sciences, 2015 Background: Clusterin is a glycoprotein that is overexpressed under stress conditions and causes cell survival by inhibiting apoptosis. Clusterin is overexpressed in prostate cancer.
N. Bakhtiari +2 more
doaj
Microbubble‐Controlled Delivery of Biofilm‐Targeting Nanoparticles to Treat MRSA Infection
Advanced Functional Materials, EarlyView.Here, an effective strategy using microbubble (MB)‐controlled delivery of biofilm‐targeting nanoparticles (BTNs) for removal and therapy of methicillin‐resistant Staphylococcus aureus (MRSA) biofilm infections is introduced. In vivo delivery of MB with BTN is demonstrated to silence key bacterial genes involved in biofilm formation (icaA), bacterial ...
Ju Yeon Chung +6 more
wiley +1 more source
CHIMIA, 1996 From efforts to improve the biophysical properties of antisense oligonucleotides by incorporating backbone- or sugar-modified nucleoside analogs, 2'-O-methoxyethyl ribonucleosides 8b were identified as building blocks for a second generation of ...
Karl-Heinz Altmann +15 more
doaj +2 more sources
Biomaterial Strategies for Targeted Intracellular Delivery to Phagocytes
Advanced Functional Materials, EarlyView.Phagocytes are essential to a functional immune system, and their behavior defines disease outcomes. Engineered particles offer a strategic opportunity to target phagocytes, harnessing inflammatory modulation in disease. By tuning features like size, shape, and surface, these systems can modulate immune responses and improve targeted treatment for a ...
Kaitlyn E. Woodworth +2 more
wiley +1 more source
Molecular Therapy: Nucleic Acids, 2017 Spinocerebellar ataxia type 3 (SCA3) is a currently incurable neurodegenerative disorder caused by a CAG triplet expansion in exon 10 of the ATXN3 gene.
Lodewijk J.A. Toonen +3 more
doaj +1 more source
Molecular Therapy: Nucleic Acids, 2023 Trigger-activatable antisense oligonucleotides have been widely applied to regulate gene function. Among them, caged cyclic antisense oligonucleotides (cASOs) maintain a specific topology that temporarily inhibits their interaction with target genes.
Zhongyu Wang +6 more
doaj +1 more source