Taking control of gene expression with light-activated oligonucleotides
BioTechniques, 2007 The recent development of caged oligonucleotides that are efficiently activated by ultraviolet (UV) light creates opportunities for regulating gene expression with very high spatial and temporal resolution.
Ivan J. Dmochowski, XinJing Tang
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Repair of Aberrant Splicing in Growth Hormone Receptor by Antisense Oligonucleotides Targeting the Splice Sites of a Pseudoexon [PDF]
, 2010 Context: The GH receptor (GHR) pseudoexon 6 Psi defect is a frequent cause of GH insensitivity (GHI) resulting from a non-functioning GH receptor (GHR). It results in a broad range of phenotypes and may also be present in patients diagnosed as idiopathic
Clark, AJL +4 more
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Site Selective Antibody-Oligonucleotide Conjugation via Microbial Transglutaminase. [PDF]
, 2019 Nucleic Acid Therapeutics (NATs), including siRNAs and AntiSense Oligonucleotides (ASOs), have great potential to drug the undruggable genome. Targeting siRNAs and ASOs to specific cell types of interest has driven dramatic improvement in efficacy and ...
Cui, Xianshu +6 more
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Molecular Therapy: Nucleic Acids, 2019 Ebola virus is the causative agent of Ebola virus disease, a severe, often fatal illness in humans. So far, there are no US Food and Drug Administration (FDA)-approved therapeutics directed against Ebola virus.
Anne Sadewasser +9 more
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Identification of antisense nucleic acid hybridization sites in mRNA molecules with self-quenching fluorescent reporter molecules [PDF]
, 2005 We describe a physical mRNA mapping strategy employing fluorescent self-quenching reporter molecules (SQRMs) that facilitates the identification of mRNA sequence accessible for hybridization with antisense nucleic acids in vitro and in vivo, real time ...
Do, Bao T. +11 more
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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
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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
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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 +3 more
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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 +51 more
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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 +5 more
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