Results 31 to 40 of about 68,586 (284)

Reconfigurable Nucleic Acid Nanoparticles with Therapeutic RNAi Responses to Intracellular Disease Markers

open access: yesAdvanced 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

Microbubble‐Controlled Delivery of Biofilm‐Targeting Nanoparticles to Treat MRSA Infection

open access: yesAdvanced 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

Chimeric Antisense Oligonucleotide Conjugated to α-Tocopherol

open access: yesMolecular Therapy: Nucleic Acids, 2015
We developed an efficient system for delivering short interfering RNA (siRNA) to the liver by using α-tocopherol conjugation. The α-tocopherol–conjugated siRNA was effective and safe for RNA interference–mediated gene silencing in vivo. In contrast, when
Tomoko Nishina   +11 more
doaj   +1 more source

Biomaterial Strategies for Targeted Intracellular Delivery to Phagocytes

open access: yesAdvanced 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

Nonviral delivery systems for antisense oligonucleotide therapeutics

open access: yesBiomaterials Research, 2022
Antisense oligonucleotides (ASOs) are an important tool for the treatment of many genetic disorders. However, similar to other gene drugs, vectors are often required to protect them from degradation and clearance, and to accomplish their transport in ...
Si Huang   +5 more
doaj   +1 more source

Molecular Mechanisms of Antisense Oligonucleotides [PDF]

open access: yesNucleic 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]

open access: yesEuropean 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

Recent Applications of Mesoporous Silica Nanoparticles in Gene Therapy

open access: yesAdvanced Healthcare Materials, EarlyView.
The review summarizes the synthesis of mesoporous silica nanoparticles (MSNs) with modifiable surface properties, functionalization strategies, mechanism of therapeutic payload release, and current applications in gene therapy, focusing on their capabilities in the targeted delivery of therapeutic nucleic acids, CRISPR‐Cas systems, and other genetic ...
Tamanna Binte Huq   +4 more
wiley   +1 more source

NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy

open access: yesMolecular Therapy: Nucleic Acids, 2018
Duchenne muscular dystrophy (DMD), the most common lethal heritable childhood disease, is caused by mutations in the DMD gene that result in the absence of functional dystrophin protein.
Naoki Watanabe   +8 more
doaj  

Targeting several CAG expansion diseases by a single antisense oligonucleotide. [PDF]

open access: yesPLoS ONE, 2011
To date there are 9 known diseases caused by an expanded polyglutamine repeat, with the most prevalent being Huntington's disease. Huntington's disease is a progressive autosomal dominant neurodegenerative disorder for which currently no therapy is ...
Melvin M Evers   +7 more
doaj   +1 more source

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