Results 21 to 30 of about 270,275 (243)
Nature Medicine, 2021 Chronic infection with hepatitis B virus (HBV) leads to an increased risk of death from cirrhosis and hepatocellular carcinoma. Functional cure rates are low with current treatment options (nucleos(t)ide analogs (NAs) and pegylated interferons ...
M. Yuen +16 more
semanticscholar +1 more source
In-silico study of antisense oligonucleotide antibiotics [PDF]
PeerJ, 2023 Background The rapid emergence of antibiotic-resistant bacteria directly contributes to a wave of untreatable infections. The lack of new drug development is an important driver of this crisis.
Erica S. Chen, Eric S. Ho
doaj +2 more sources
SUPPRESSION OF MUTANT C9ORF72 EXPRESSION BY A POTENT MIXED BACKBONE ANTISENSE OLIGONUCLEOTIDE
Nature Medicine, 2021 Expansions of a G4C2 repeat in the C9ORF72 gene are the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), two devastating adult-onset neurodegenerative disorders.
Hélène Tran +20 more
semanticscholar +1 more source
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 +4 more
doaj +1 more source
The Challenges and Strategies of Antisense Oligonucleotide Drug Delivery
Biomedicines, 2021 Antisense oligonucleotides (ASOs) are used to selectively inhibit the translation of disease-associated genes via Ribonuclease H (RNaseH)-mediated cleavage or steric hindrance.
M. Gagliardi, A. T. Ashizawa
semanticscholar +1 more source
Nature Medicine, 2021 Leber congenital amaurosis due to CEP290 ciliopathy is being explored by treatment with the antisense oligonucleotide (AON) sepofarsen. One patient who was part of a larger cohort (ClinicalTrials.gov NCT03140969) was studied for 15 months after a single ...
A. Cideciyan +9 more
semanticscholar +1 more source
Antisense oligonucleotide therapy for neurodevelopmental disorders
Developmental Neuroscience, 2021 Antisense oligonucleotides (ASOs) are short oligonucleotides that can modify gene expression and mRNA splicing in the nervous system. The FDA has approved ASOs for treatment of ten genetic disorders, with many applications currently in the pipeline.
Sophie F. Hill, M. Meisler
semanticscholar +1 more source
Polymerase-endonuclease amplification reaction (PEAR) for large-scale enzymatic production of antisense oligonucleotides. [PDF]
PLoS ONE, 2010 Antisense oligonucleotides targeting microRNAs or their mRNA targets prove to be powerful tools for molecular biology research and may eventually emerge as new therapeutic agents.
Xiaolong Wang +2 more
doaj +1 more source
History of Research on Antisense Oligonucleotide Analogs
Substantia, 2021 In the search for novel therapeutics, antisense oligonucleotide (ASO) analogs have been a major focus of research for over 40 years. They use the antisense strategy, namely they have a nucleic acid base sequence that is complementary to a portion of a ...
Jack S. Cohen
doaj +1 more source
RNA Interference-Guided Targeting of Hepatitis C Virus Replication with Antisense Locked Nucleic Acid-Based Oligonucleotides Containing 8-oxo-dG Modifications. [PDF]
PLoS ONE, 2015 The inhibitory potency of an antisense oligonucleotide depends critically on its design and the accessibility of its target site. Here, we used an RNA interference-guided approach to select antisense oligonucleotide target sites in the coding region of ...
Margit Mutso +10 more
doaj +1 more source