Androgen Receptor Antisense Oligonucleotide AZD5312 [PDF]
National Cancer Institute
openalex +1 more source
The how and why of lncRNA function: An innate immune perspective. [PDF]
Next-generation sequencing has provided a more complete picture of the composition of the human transcriptome indicating that much of the "blueprint" is a vastness of poorly understood non-protein-coding transcripts.
Carpenter, Susan +2 more
core
Targeting RyR Activity Boosts Antisense Exon 44 and 45 Skipping in Human DMD Skeletal or Cardiac Muscle Culture Models. [PDF]
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
core +1 more source
Protein complexes like KIBRA‐PKMζ are crucial for maintaining memories, forming month‐long protein traces in memory‐tagged neurons, but conventional RNA‐seq analysis fails to detect their transcript changes, leaving memory molecules undetected in the shadows of abundantly‐expressed genes.
Jiyeon Han +10 more
wiley +1 more source
Rethinking antisense oligonucleotide therapeutics for amyotrophic lateral sclerosis
Antisense oligonucleotides, which are used to silence target genes, are gaining attention as a novel drug discovery modality for proteinopathies. However, while clinical trials for neurodegenerative diseases like amyotrophic lateral sclerosis have been ...
Daisuke Ito, Kensuke Okada
doaj +1 more source
First Generation Proteolysis Targeting Chimeras (PROTACs) for the Treatment of Progeria
We report the first PROTACs designed to degrade progerin, introducing a novel therapeutic approach for progeria. The best compound, UCM‐18142, significantly reduces progerin levels and improves key disease phenotypes in patient‐derived cells and in the LmnaG609G/G609G mouse model, paving the way for new treatment strategies targeting the root cause of ...
Jon Macicior‐Michelena +5 more
wiley +1 more source
Antisense oligonucleotides and their applications in rare neurological diseases
Rare diseases affect almost 500 million people globally, predominantly impacting children and often leading to significantly impaired quality of life and high treatment costs. While significant contributions have been made to develop effective treatments
Simon McDowall +7 more
doaj +1 more source
Inhibition of PaCaMKII-E isoform in the dorsal unpaired median neurosecretory cells of cockroach reduces nicotine- and clothianidin-induced currents [PDF]
Cellular responses to Ca(2+) require intermediary proteins such as calcium/calmodulin-dependent protein kinase II (CaMKII), which transduces the signal into downstream effects.
D. Calas-List +5 more
core +4 more sources
Hepatic HDGF as a key mediator in coordinating hepatic steatosis and intrahepatic crosstalk in MASLD. Activation of HDGF facilitates its interaction with both STAT3 and S6K1, driving the S6K1‐dependent STAT3 phosphorylation and subsequently enhancing hepatic lipogenesis.
Jian Wen +28 more
wiley +1 more source
Antisense oligonucleotide–mediated inhibition of angiopoietin-like protein 3 increases reverse cholesterol transport in mice [PDF]
Thomas A. Bell +5 more
openalex +1 more source

