Micro-RNAS that control myosin expression and myofiber identity [PDF]
, 2015 The present invention relates to the identification of two microRNAs, miR-499 and miR-208b, that repress fast skeletal muscle contractile protein genes. Expression of miR-499 and/or miR-208b can be used to repress fast fiber genes and activate slow fiber
Olson, Eric N., van Rooij, Eva
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Antisense oligonucleotides for neurodegeneration [PDF]
Science, 2020 Promising clinical results for Huntington's disease give hope for other ...
Leavitt, BR, Tabrizi, SJ
openaire +3 more sources
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
doaj +1 more source
Targeting STAT3 in Cancer with Nucleotide Therapeutics. [PDF]
, 2019 Signal transducer and activator of transcription 3 (STAT3) plays a critical role in promoting the proliferation and survival of tumor cells. As a ubiquitously-expressed transcription factor, STAT3 has commonly been considered an "undruggable" target for ...
Grandis, Jennifer R +3 more
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Identification of sequence motifs significantly associated with antisense activity
BMC Bioinformatics, 2007 Background Predicting the suppression activity of antisense oligonucleotide sequences is the main goal of the rational design of nucleic acids. To create an effective predictive model, it is important to know what properties of an oligonucleotide ...
Peek Andrew S, McQuisten Kyle A
doaj +1 more source
How late is too late for treatment?
eLife, 2023 Experiments on mice suggest that an approach called antisense oligonucleotide therapy may be able to treat some symptoms of Angelman syndrome, including problems with epilepsy and sleep.
Lawrence T Reiter
doaj +1 more source
C9orf72 repeat expansions cause neurodegeneration in Drosophila through arginine-rich proteins [PDF]
, 2014 An expanded GGGGCC repeat in C9orf72 is the most common genetic cause of frontotemporal dementia and amyotrophic lateral sclerosis. A fundamental question is whether toxicity is driven by the repeat RNA itself and/or by dipeptide repeat proteins ...
Cabecinha, M +19 more
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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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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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Targeting RyR Activity Boosts Antisense Exon 44 and 45 Skipping in Human DMD Skeletal or Cardiac Muscle Culture Models. [PDF]
, 2019 Systemic delivery of antisense oligonucleotides (AO) for DMD exon skipping has proven effective for reframing DMD mRNA, rescuing dystrophin expression, and slowing disease progression in animal models.
Barthélémy, Florian +6 more
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