Results 31 to 40 of about 119,501 (270)

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

Biochemical prevention and treatment of viral infections – A new paradigm in medicine for infectious diseases [PDF]

, 2004
For two centuries, vaccination has been the dominating approach to develop prophylaxis against viral infections through immunological prevention. However, vaccines are not always possible to make, are ineffective for many viral infections, and also carry
Fang, Fang, Le Calvez, Hervé, Yu, Mang
core   +3 more sources

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

Inhibition of Nonsense-Mediated mRNA Decay by Antisense Morpholino Oligonucleotides Restores Functional Expression of hERG Nonsense and Frameshift Mutations in Long-QT Syndrome

, 2010
Mutations in the human ether-a-go-go-related gene (hERG) cause long-QT syndrome type 2 (LQT2). We previously described a homozygous LQT2 nonsense mutation Q1070X in which the mutant mRNA is degraded by nonsense-mediated mRNA decay (NMD) leading to a ...
Bhuiyan, Bhuiyan, Conti, Curran, Dominski, Friedman, Frischmeyer, Gong, Gong, Gritz, Holbrook, Howard, Jearawiriyapaisarn, Kapplinger, Khajavi, Kuzmiak, Le Hir, Le Roy, Linde, Lykke-Andersen, Matthew R. Stump, Millat, Nagy, Napolitano, Nicholson, Qiuming Gong, Sanguinetti, Schwartz, Shimizu, Splawski, Stalder, Tester, Trudeau, Usuki, Wu, Yao, Zhang, Zhengfeng Zhou, Zhou, Zhou   +39 more
core   +1 more source

Silencing disease genes in the laboratory and the clinic

, 2011
Synthetic nucleic acids are commonly used laboratory tools for modulating gene expression and have the potential to be widely used in the clinic. Progress towards nucleic acid drugs, however, has been slow and many challenges remain to be overcome before
Corey, David R., Watts, Jonathan K.
core   +1 more source

Micro-RNA family that modulates fibrosis and uses thereof [PDF]

, 2017
The present invention relates to the identification of a microRNA family, designated miR-29a-c, that is a key regulator of fibrosis in cardiac tissue. The inventors show that members of the miR-29 family are down-regulated in the heart tissue in response
Olson, Eric N., van Rooij, Eva
core   +1 more source

Steric antisense inhibition of AMPA receptor Q/R editing reveals tight coupling to intronic editing sites and splicing [PDF]

, 2012
Adenosine-to-Inosine (A-to-I) RNA editing is a post-transcriptional mechanism, evolved to diversify the transcriptome in metazoa. In addition to wide-spread editing in non-coding regions protein recoding by RNA editing allows for fine tuning of protein ...
Ales Balik, Altschul, Andrew C. Penn, Arzumanov, Arzumanov, Balik, Bass, Birney, Brusa, Carver, Daniel, Darty, Dominski, Egebjerg, Eisen, Enstero, Feldmeyer, Gallo, Ge, Greger, Hamada, Heasman, Higuchi, Higuchi, Hogg, Ingo H. Greger, Jepson, Jubin, Katoh, Kent, Kwak, Laurencikiene, Maas, Moulton, Peng, Reenan, Rice, Rosenthal, Rueter, Ryman, Schoft, Sommer, Stephens, Summerton, Summerton, Summerton, Tariq, Traynelis, Valente, Venkatesh, Yamashita, Yang   +51 more
core   +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