Results 31 to 40 of about 218,639 (188)

Structural Diversity of Sense and Antisense RNA Hexanucleotide Repeats Associated with ALS and FTLD

Molecules, 2020
The hexanucleotide expansion GGGGCC located in C9orf72 gene represents the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal lobar dementia (FTLD).
Tim Božič, Matja Zalar, Boris Rogelj, Janez Plavec, Primož Šket   +4 more
doaj   +1 more source

A Kinetic Model Explains Why Shorter and Less Affine Enzyme-recruiting Oligonucleotides Can Be More Potent

Molecular Therapy: Nucleic Acids, 2014
Antisense oligonucleotides complementary to RNA targets promise generality and ease of drug design. The first systemically administered antisense drug was recently approved for treatment and others are in clinical development. Chemical modifications that
Lykke Pedersen, Peter H Hagedorn, Marie Wickström Lindholm, Morten Lindow   +3 more
doaj   +1 more source

In vivo uptake of antisense oligonucleotide drugs predicted by ab initio quantum mechanical calculations

Scientific Reports, 2021
Liver and kidney uptake and antisense activity is studied for a series of Locked Nucleic Acid (LNA) oligonucleotides with fully stereo-defined, internucleoside linkages.
Henrik Frydenlund Hansen, Nanna Albaek, Bo Rode Hansen, Irene Shim, Henrik Bohr, Troels Koch   +5 more
doaj   +1 more source

Antisense Oligonucleotides: Concepts and Pharmaceutical Applications

Borneo Journal of Pharmacy, 2023
Antisense oligonucleotides are drugs whose mechanism is based on binding to RNA target sequences. For this purpose, they modify the protein expression through steric hindrance and exon omission.
Ariana Araya, David Arias, Karla Coto, María Rebeca López, Joselyn Rivera, Juan José Mora   +5 more
doaj   +1 more source

Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models

Journal of Clinical Investigation, 2018
Mutations in superoxide dismutase 1 (SOD1) are responsible for 20% of familial ALS. Given the gain of toxic function in this dominantly inherited disease, lowering SOD1 mRNA and protein is predicted to provide therapeutic benefit.
A. McCampbell, Tracy A. Cole, A. Wegener, G. S. Tomassy, Amy Setnicka, B. Farley, Kathleen M. Schoch, Mariah L. Hoye, Mark Shabsovich, Linhong Sun, Yi Luo, Mingdi Zhang, Nicole Comfort, Bin Wang, Jessica Amacker, S. Thankamony, D. Salzman, M. Cudkowicz, Danielle L. Graham, C. Bennett, H. Kordasiewicz, E. Swayze, T. Miller   +22 more
semanticscholar   +1 more source

Silencing Antibiotic Resistance with Antisense Oligonucleotides

Biomedicines, 2021
Antisense technologies consist of the utilization of oligonucleotides or oligonucleotide analogs to interfere with undesirable biological processes, commonly through inhibition of expression of selected genes.
Saumya Jani, Maria Soledad Ramirez, Marcelo E. Tolmasky   +2 more
doaj   +1 more source

Targeted delivery of antisense oligonucleotides to pancreatic β-cells

Science Advances, 2018
Receptor-dependent productive uptake of GLP1-conjugated antisense oligonucleotides occurs selectively in pancreatic β-cells. Antisense oligonucleotide (ASO) silencing of the expression of disease-associated genes is an attractive novel therapeutic ...
Carina Ämmälä, W. Drury, L. Knerr, I. Ahlstedt, P. Stillemark-Billton, C. Wennberg-Huldt, E. Andersson, E. Valeur, R. Jansson-Löfmark, D. Janzén, Linda Sundström, J. Meuller, J. Claesson, P. Andersson, C. Johansson, R. Lee, T. P. Prakash, P. Seth, B. Monia, Shalini Andersson   +19 more
semanticscholar   +1 more source

Transport Oligonucleotides—A Novel System for Intracellular Delivery of Antisense Therapeutics

Molecules, 2020
Biological activity of antisense oligonucleotides (asON), especially those with a neutral backbone, is often attenuated by poor cellular accumulation. In the present proof-of-concept study, we propose a novel delivery system for asONs which implies the ...
Oleg V. Markov, Anton V. Filatov, Maxim S. Kupryushkin, Ivan V. Chernikov, Olga A. Patutina, Anton A. Strunov, Elena L. Chernolovskaya, Valentin V. Vlassov, Dmitrii V. Pyshnyi, Marina A. Zenkova   +9 more
doaj   +1 more source

Efficient inhibition of HIV-1 expression by LNA modified antisense oligonucleotides and DNAzymes targeted to functionally selected binding sites

Retrovirology, 2007
Background A primary concern when targeting HIV-1 RNA by means of antisense related technologies is the accessibility of the targets. Using a library selection approach to define the most accessible sites for 20-mer oligonucleotides annealing within the ...
Berkhout Ben, Wengel Jesper, Haasnoot Joost, Jakobsen Martin R, Kjems Jørgen   +4 more
doaj   +1 more source

Receptor-Mediated Uptake of Phosphorothioate Antisense Oligonucleotides in Different Cell Types of the Liver.

Nucleic Acid Therapeutics, 2018
Oligonucleotide therapeutics have emerged as a third distinct platform for drug discovery within the pharmaceutical industry. Five oligonucleotide-based drugs have been approved by the US FDA and over 100 oligonucleotides drugs are currently at different
Colton M. Miller, M. Tanowitz, A. J. Donner, T. P. Prakash, E. Swayze, E. Harris, P. Seth   +6 more
semanticscholar   +1 more source