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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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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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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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-RNAs of the miR-15 family modulate cardiomyocyte survival and cardiac repair [PDF]
, 2015 A family of microRNAs, called the miR-15 family, which includes miR-195, are shown to be up-regulated during pathological cardiac remodeling and repress the expression of mRNAs required for cell proliferation and survival, with consequent loss of ...
Olson, Eric N., van Rooij, Eva
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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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Antisense-mediated exon skipping: a therapeutic strategy for titin-based dilated cardiomyopathy [PDF]
, 2015 Frameshift mutations in the TTN gene encoding titin are a major cause for inherited forms of dilated cardiomyopathy (DCM), a heart disease characterized by ventricular dilatation, systolic dysfunction, and progressive heart failure. To date, there are no
Alessandra, Moretti +22 more
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HLA-G: expression in human keratinocytes in vitro and in human skin in vivo [PDF]
, 1994 Classical, polymorphic major histocompatibility complex class I molecules are expressed on most nucleated cells.They present peptides at the cell surface and, thus, enable the immune system to scan peptides for their antigenicity.
Ulbrecht, M. +7 more
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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 +5 more
doaj +1 more source
Evidence Favoring a Positive Feedback Loop for Physiologic Auto Upregulation of hnRNP-E1 during Prolonged Folate Deficiency in Human Placental Cells [PDF]
, 2017 Background: Previously, we determined that heterogeneous nuclear ribonucleoprotein E1 (hnRNP-E1) functions as an intracellular physiologic sensor of folate deficiency.
Antony, Aśok C. +7 more
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