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Antisense oligonucleotides [PDF]

Neurology Genetics, 2019
There are few disease-modifying therapeutics for neurodegenerative diseases, but successes on the development of antisense oligonucleotide (ASO) therapeutics for spinal muscular atrophy and Duchenne muscular dystrophy predict a robust future for ASOs in medicine.
Scoles, Daniel R., Minikel, Eric V., Pulst, Stefan M.   +2 more
openaire   +3 more sources

Ultrasmall nanoparticles for co-delivery of antisense oligonucleotides targeting miR-21 and miR-210 to treat glioblastoma [PDF]

Journal of Nanobiotechnology
Glioblastoma (GBM) is the most common and aggressive type of brain tumour, with less than 5% of patients surviving more than 5 years. Despite decades of research to understand the underlying pathophysiological causes, it has witnessed very slow progress ...
Ravi Raj Singh, Ritu Kulshreshtha, Amirali Popat   +2 more
doaj   +2 more sources

Splice-Switching Antisense Oligonucleotides Targeting Extra- and Intracellular Domains of Epidermal Growth Factor Receptor in Cancer Cells

Biomedicines, 2023
Cancer is one of the leading causes of death globally. Epidermal growth factor receptor is one of the proteins involved in cancer cell proliferation, differentiation, and invasion. Antisense oligonucleotides are chemical nucleic acids that bind to target
Akilandeswari Ashwini Balachandran, Prithi Raguraman, Kamal Rahimizadeh, Rakesh N. Veedu   +3 more
doaj   +1 more source

Antisense oligonucleotides: recent progress in the treatment of various diseases

Beni-Suef University Journal of Basic and Applied Sciences, 2022
Background Antisense oligonucleotides are a promising novel class of therapeutic agents to treat different diseases in living things. They provide an efficient method for making target-selective agents because they change gene expression sequences ...
Chandravadivelu Gopi, Magharla Dasaratha Dhanaraju, Kavitha Dhanaraju   +2 more
doaj   +1 more source

Effect of native gastric mucus on in vivo hybridization therapies directed at Helicobacter pylori [PDF]

, 2015
Helicobacter pylori infects more than 50% of the worldwide population. It is mostly found deep in the gastric mucus lining of the stomach, being a major cause of peptic ulcers and gastric adenocarcinoma. To face the increasing resistance of H.
Azevedo, Nuno F, Braeckmans, Kevin, Dakwar, George, De Smedt, Stefaan, Figueiredo, Céu, Fontenete, Silvia, Forier, Katrien, Guimarães, Nuno, Leite, Marina, Remaut, Katrien, Santos, Rita, Stremersch, Stephan, Wengel, Jesper, Xiong, Ranhua   +13 more
core   +16 more sources

An Investigation into the Potential of Targeting Escherichia coli rne mRNA with Locked Nucleic Acid (LNA) Gapmers as an Antibacterial Strategy

Molecules, 2021
The increase in antibacterial resistance is a serious challenge for both the health and defence sectors and there is a need for both novel antibacterial targets and antibacterial strategies.
Layla R. Goddard, Charlotte E. Mardle, Hassan Gneid, Ciara G. Ball, Darren M. Gowers, Helen S. Atkins, Louise E. Butt, Jonathan K. Watts, Helen A. Vincent, Anastasia J. Callaghan   +9 more
doaj   +1 more source

Prdm1 (Blimp-1) and the Expression of Fast and Slow Myosin Heavy Chain Isoforms during Avian Myogenesis In Vitro [PDF]

, 2010
BACKGROUND. Multiple types of fast and slow skeletal muscle fibers form during early embryogenesis in vertebrates. In zebrafish, formation of the earliest slow myofibers in fin muscles requires expression of the zinc-finger transcriptional repressor ...
Ardelt, Magdalena, Beermann, Mary Lou, Girgenrath, Mahasweta, Miller, Jeffrey Boone   +3 more
core   +14 more sources

Accessible light-controlled knockdown of cell-free protein synthesis using phosphorothioate-caged antisense oligonucleotides

Communications Chemistry, 2023
Light-activated antisense oligonucleotides have been developed to induce gene knockdown in living cells, however, their synthesis remains challenging and application in cell-free systems is underexplored.
Denis Hartmann, Michael J. Booth
doaj   +1 more source

Nucleobase-modified antisense oligonucleotides containing 5-(phenyltriazol)-2′-deoxyuridine nucleotides induce exon-skipping:In vitro [PDF]

, 2017
We investigated the potential of nucleobase-modified antisense oligonucleotides to induce exon-skipping, and found that 5-(phenyltriazol)-2′-deoxyuridine-modified antisense oligonucleotides induced efficient exon-skipping in vitro.
Hornum, Mick, Le, Bao T., Nielsen, Poul, Sharma, Pawan K., Veedu, Rakesh N.   +4 more
core   +1 more source

Synthesis and properties of oligonucleotides modified with an N-methylguanidine-bridged nucleic acid (GuNA[Me]) bearing adenine, guanine, or 5-methylcytosine nucleobases

Beilstein Journal of Organic Chemistry, 2021
Chemical modifications have been extensively used for therapeutic oligonucleotides because they strongly enhance the stability against nucleases, binding affinity to the targets, and efficacy. We previously reported that oligonucleotides modified with an
Naohiro Horie, Takao Yamaguchi, Shinji Kumagai, Satoshi Obika   +3 more
doaj   +1 more source