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Molecular Therapy: Nucleic Acids
Scopus
ISSN 2162-2531 · Elsevier · 2.929 artikel di indeks
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Artikel Jurnal
Identification and diagnostic evaluation of an aptamer targeting prostate-cancer-derived small extracellular vesicles
Prostate cancer (PCa) lacks convenient, non-invasive, and highly specific diagnostic markers. Aptamers have emerged as preferred probes for biosensors that target extracellular vesicles (EVs). This study aimed to explore the diagnostic value of PCa-specific EVs aptamer probes. We used EV-SELEX to…
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Artikel Jurnal
Improving angiogenesis ameliorates the efficacy of ASO-based exon skipping for the treatment of Duchenne muscular dystrophy
Duchenne muscular dystrophy (DMD) is a severe X-linked disease caused by pathogenic variants in the DMD gene, resulting in the absence of functional dystrophin. Antisense oligonucleotide (ASO)-based therapies aim to restore the open reading frame and produce a truncated but functional dystrophin…
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Artikel Jurnal
Identification and functional validation of intracellular protein partners of phosphorothioate splice-switching oligonucleotides using AP-MS
Cellular uptake and intracellular distribution of phosphorothioate-modified antisense oligonucleotides (PS-ASOs) are mediated by protein interactions. While several PS-ASOs-binding proteins have been identified, mainly using gapmer designs with 2′-O-methoxyethyl (2′MOE) modifications, less is known…
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Artikel Jurnal
Engineered miR-122 inhibitors preserve endothelial mitochondrial function and prevent vascular dysfunction in obesity-associated prediabetes
MicroRNA-122-5p (miR-122) is primarily expressed in the liver and is increasingly released into the bloodstream during obesity. It impacts the function of non-liver tissues, such as vascular endothelial cells, and increases the risk of diabetic vasculopathy. Using a gamma-peptide-nucleic acid-based…
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c-Jun in neurodegeneration: A key transcriptional regulator with therapeutic implications
c-Jun, a core component of the activating protein-1 (AP-1) transcription factor complex, regulates cellular processes including proliferation, differentiation, survival, apoptosis, and oncogenesis. c-Jun functions by dimerizing to bind DNA and modulates the expression of genes such as Bcl-2, cyclin…
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Artikel Jurnal
Optimizing C14120-based LNPs for in vitro and in vivo mRNA delivery
Lipid nanoparticles (LNPs) have proven to be an effective delivery system for RNA therapeutics. The chemical composition of LNPs determines their functional delivery efficiency and targeting properties, which vary between in vitro and in vivo contexts. Here, we have systematically characterized and…
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Downregulation of S100a8 mitigates fibrosis in skeletal muscles affected by Marfan syndrome
This study investigated the potential of eccentric training in attenuating the deleterious effects of Marfan syndrome on skeletal muscles. Marfan syndrome and wild-type mice were divided into a trained and a control group; the former performed a downhill running protocol for 8 weeks. Morphological…
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RNA-based discovery and correction of splicing defects caused by POLR3A missense mutations
RNA polymerase III-related disorders represent a clinically diverse spectrum of diseases. While pathogenic variants in the POLR3A subunit are most commonly associated with hypomyelinating leukodystrophies, they also contribute to other neurological or systemic deficiencies. Despite this phenotypic…
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Generation of aptamers for the selective detection and neutralization of soluble lymphotoxin alpha
Lymphotoxin alpha (LTα) is a potent inflammatory cytokine implicated in the pathophysiology of numerous human autoimmune and inflammatory diseases. Existing as a soluble homotrimer (LTα3) or membrane-bound heterotrimer (LTα1β2), the differential and distinct functions of lymphotoxin signaling have…
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Artikel Jurnal
microRNA-422a promotes HIV replication and innate immune evasion by targeting MECP2
Understanding the mechanisms underlying interferon-alpha (IFNα) anti-human immunodeficiency virus type I (HIV-1) effects and HIV-1 innate immune evasion is critical to developing improved antiviral strategies. We previously reported that the microRNA (miRNA) miR-422a was the sole miRNA…
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