Results 81 to 90 of about 266,614 (380)

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, Erik S.G. Stroes, John J.P. Kastelein, Maartje E. Visser   +3 more
openaire   +3 more sources

Membrane Fusion‐Inspired Nanomaterials: Emerging Strategies for Infectious Disease and Cancer Diagnostics

Advanced Healthcare Materials, EarlyView.
Membrane fusion‐inspired nanomaterials offer transformative potential in diagnostics by mimicking natural fusion processes to achieve highly sensitive and specific detection of disease biomarkers. This review highlights recent advancements in nanomaterial functionalization strategies, signal amplification systems, and stimuli‐responsive fusion designs,
Sojeong Lee, Khulan Nyamzaya, Jueun Han, Yejin Song, Jihee Lee, Jung Hyun Jo, Seungmin Bang, Eun‐Kyung Lim, Seungjoo Haam, Eunjung Kim   +9 more
wiley   +1 more source

DNA Barcoding‐Enabled Tracking of Lipid Nanoparticles: Drug‐Loading‐Dependent Biodistribution and Tumor Microenvironment Targeting

Advanced Healthcare Materials, EarlyView.
In this study, lipid nanoparticles are tracked in vivo using DNA barcodes to determine how varying drug loadings affect their delivery behaviors in mice. A cost‐effective strategy is developed for simultaneously monitoring the biodistribution of a library of lipid nanoparticles (LNPs).
Letao Xu, Rui Chen, Xing Wang, Dawei Liu, Yun liu, Chun‐Xia Zhao   +5 more
wiley   +1 more source

Antisense oligonucleotide-mediated ataxin-1 reduction prolongs survival in SCA1 mice and reveals disease-associated transcriptome profiles.

JCI Insight, 2018
Spinocerebellar ataxia type 1 (SCA1) is a dominantly inherited ataxia caused by expansion of a translated CAG repeat encoding a glutamine tract in the ataxin-1 (ATXN1) protein.
Jillian Friedrich, H. Kordasiewicz, B. O'Callaghan, Hillary P. Handler, C. Wagener, L. Duvick, E. Swayze, Orion Rainwater, Bente Hofstra, M. Benneyworth, Tessa Nichols-Meade, Praseuth Yang, Zhao Chen, Judit M. Pérez Ortiz, H. Clark, G. Öz, Sarah N. Larson, H. Zoghbi, C. Henzler, H. Orr   +19 more
semanticscholar   +1 more source

The Potential Protective Effect and Underlying Mechanisms of Physiological Unconjugated Hyperbilirubinemia Mediated by UGT1A1 Antisense Oligonucleotide Therapy in a Mouse Model of Cyclosporine A-Induced Chronic Kidney Disease

Metabolites, 2022
Cyclosporine A (CSA) is an immunosuppressive drug that has improved transplant survival rates. However, its use is often limited because it is thought to be linked to the development of chronic kidney disease after kidney transplants. This study aimed to
Basma H. Marghani, Mohamed El-Adl, Ahmed I. Ateya, Basma H. Othman, Heba I. Ghamry, Mustafa Shukry, Mohamed Mohamed Soliman, Mohamed Abdo Rizk   +7 more
doaj   +1 more source

Suppressing t(4;11) Acute Leukemia by Lipopolymer Nanoparticle Delivery of siRNA Targeting KMT2A::AFF1 with Enhanced Extrahepatic Delivery

Advanced Healthcare Materials, EarlyView.
This study introduces a new lipopolymer nanoparticle (LPNP) system that efficiently delivers siRNA to leukemia cells. The LPNPs silence the leukemia fusion gene KMT2A::AFF1, induce apoptosis, and decrease leukemia burden in mice. These results demonstrate the potential of LPNPs as a targeted siRNA therapy for acute lymphoblastic leukemia.
Mohammad Nasrullah, Remant KC, Amarnath Praphakar Rajendran, Saba Abbasi Dezfouli, Cezary Kucharski, Xiaoyan Jiang, Spencer B. Gibson, Joseph Brandwein, Olaf Heidenreich, Hasan Uludağ   +9 more
wiley   +1 more source

The Dynamics of Compound, Transcript, and Protein Effects After Treatment With 2OMePS Antisense Oligonucleotides in mdx Mice

Molecular Therapy: Nucleic Acids, 2014
Antisense-mediated exon skipping is currently in clinical development for Duchenne muscular dystrophy (DMD) to amend the consequences of the underlying genetic defect and restore dystrophin expression. Due to turnover of compound, transcript, and protein,
Ingrid E C Verhaart, Laura van Vliet-van den Dool, Jessica A Sipkens, Sjef J de Kimpe, Ingrid G M Kolfschoten, Judith C T van Deutekom, Lia Liefaard, Jim E Ridings, Steve R Hood, Annemieke Aartsma-Rus   +9 more
doaj   +1 more source

Correction of Clcn1 alternative splicing reverses muscle fiber type transition in mice with myotonic dystrophy

Nature Communications, 2023
In a double homozygous mouse model of myotonic dystrophy type 1, Hu et al. use antisense oligonucleotide correction of myotonia to induce a therapeutic shift from an overabundance of oxidative muscle fibers to mechanically stronger glycolytic fibers.
Ningyan Hu, Eunjoo Kim, Layal Antoury, Thurman M. Wheeler   +3 more
doaj   +1 more source

Snail2 directly represses cadherin6B during epithelial-to-mesenchymal transitions of the neural crest [PDF]

, 2007
The neural crest, a transient population of migratory cells, forms the craniofacial skeleton and peripheral nervous system, among other derivatives in vertebrate embryos. The transcriptional repressor Snail2 is thought to be crucial for the epithelial-to-
Bronner-Fraser, Marianne, Coles, Edward G., Taneyhill, Lisa A.   +2 more
core   +1 more source

Tegsedi (Inotersen): An Antisense Oligonucleotide Approved for the Treatment of Adult Patients with Hereditary Transthyretin Amyloidosis

Pharmaceuticals, 2019
Tegsedi (Inotersen) is a chemically modified antisense oligonucleotide that inhibits the hepatic production of transthyretin (TTR). Several single-point mutations in TTR destabilize its structure, leading to the aggregation and accumulation of amyloid ...
L. Gales
semanticscholar   +1 more source