Results 61 to 70 of about 270,275 (243)

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

Synthesis and Antisense Properties of 2′β-F-Arabinouridine Modified Oligonucleotides with 4′-C-OMe Substituent

Molecules, 2018
A novel 2′-F,4′-C-OMe–arabinouridine (araU) was successfully synthesized and introduced into oligonucleotides. The oligonucleotide containing 2′-F,4′-C-OMe–araU exhibited improved nuclease resistance and RNA ...
Xiao-Yang He, Jing Wang, Dan-Dan Lu, Sheng-Qi Wang   +3 more
doaj   +1 more source

Chimeric Antisense Oligonucleotide Conjugated to α-Tocopherol

Molecular 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, Junna Numata, Kazutaka Nishina, Kie Yoshida-Tanaka, Keiko Nitta, Wenying Piao, Rintaro Iwata, Shingo Ito, Hiroya Kuwahara, Takeshi Wada, Hidehiro Mizusawa, Takanori Yokota   +11 more
doaj   +1 more source

Antisense Oligonucleotide: Basic Concepts and Therapeutic Application in Inflammatory Bowel Disease

Frontiers in Pharmacology, 2019
Several molecular technologies aimed at regulating gene expression that have been recently developed as a strategy to combat inflammatory and neoplastic diseases.
D. Di Fusco, V. Dinallo, I. Marafini, M. Figliuzzi, B. Romano, G. Monteleone   +5 more
semanticscholar   +1 more source

Antisense oligonucleotide nomenclature.

, 2014
MSP, muscle-specific peptide and B - β-alanine [34].Antisense oligonucleotide nomenclature.
Ben Gu (653768), Limin Cao (407556), Gang Han (127634), HaiFang Yin (2177172)   +3 more
core   +1 more source

Chemistry, structure and function of approved oligonucleotide therapeutics

Nucleic Acids Research, 2023
Eighteen nucleic acid therapeutics have been approved for treatment of various diseases in the last 25 years. Their modes of action include antisense oligonucleotides (ASOs), splice-switching oligonucleotides (SSOs), RNA interference (RNAi) and an RNA ...
M. Egli, M. Manoharan
semanticscholar   +1 more source

A New Antisense Phosphoryl Guanidine Oligo-2′-O-Methylribonucleotide Penetrates Into Intracellular Mycobacteria and Suppresses Target Gene Expression

Frontiers in Pharmacology, 2019
The worldwide spread of multidrug-resistant Mycobacterium tuberculosis strains prompted the development of new strategies to combat tuberculosis, one of which is antisense therapy based on targeting bacterial mRNA by oligonucleotide derivatives. However,
Yulia V. Skvortsova, Elena G. Salina, Ekaterina A. Burakova, Ekaterina A. Burakova, Oksana S. Bychenko, Dmitry A. Stetsenko, Dmitry A. Stetsenko, Tatyana L. Azhikina   +7 more
doaj   +1 more source

Antisense Oligonucleotide-Mediated Transcript Knockdown in Zebrafish. [PDF]

PLoS ONE, 2015
Antisense oligonucleotides (ASOs) are synthetic, single-strand RNA-DNA hybrids that induce catalytic degradation of complementary cellular RNAs via RNase H.
Andrea Pauli, Tessa G Montague, Kim A Lennox, Mark A Behlke, Alexander F Schier   +4 more
doaj   +1 more source

Antisense oligonucleotides in neurological disorders [PDF]

Therapeutic Advances in Neurological Disorders, 2018
The introduction of genetics revolutionized the field of neurodegenerative and neuromuscular diseases and has provided considerable insight into the underlying pathomechanisms. Nevertheless, effective treatment options have been limited. This changed recently when antisense oligonucleotides (ASOs) could be translated from in vitro and experimental ...
Claudia D. Wurster, Albert C. Ludolph
openaire   +3 more sources

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, J. P. Ortiz, H. Clark, G. Öz, Sarah N. Larson, H. Zoghbi, C. Henzler, H. Orr   +19 more
semanticscholar   +1 more source