Novel Therapeutics for Familial Chylomicronemia Syndrome. [PDF]
Izar MC, Fonseca FAH.
europepmc +1 more source
MicroRNA‐100‐5p Exacerbates Myocardial Ischemia–Reperfusion Injury Through Downregulation of PRMT5
MiR‐100‐5p overexpression downregulates PRMT5, leading to increased PTEN activity and reduced AKT signaling. This molecular cascade promotes cardiomyocyte apoptosis, enhances fibrotic remodeling, and exacerbates cardiac dysfunction following myocardial ischemia–reperfusion injury.
Zhenya Wang+9 more
wiley +1 more source
Antisense oligonucleotides directed against App and Rab5 normalized endosomal Rab activity and reversed DS-AD-linked degenerative phenotypes in the Dp16 mouse model of Down syndrome. [PDF]
Chen XQ+12 more
europepmc +1 more source
APP antisense oligonucleotides are effective in rescuing mitochondrial phenotypes in human iPSC-derived trisomy 21 astrocytes. [PDF]
Thirumalai S+3 more
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Sequence-Specific Free Energy Changes in DNA/RNA Induced by a Single LNA-T Modification in Antisense Oligonucleotides. [PDF]
Tomita-Sudo E+4 more
europepmc +1 more source
Lysine demethylase 6A (KDM6A), an epigenetic regulator expressed in hematopoietic stem and progenitor cells (HSPCs), plays a critical role in hematopoietic homeostasis. Deficiency of KDM6A induces myeloid lineage skewing and drives the development of a chronic myelomonocytic leukemia (CMML)‐like disease.
Huiqiao Chen+11 more
wiley +1 more source
Targeting miRNA‐1a and miRNA‐15b: A Novel Combinatorial Strategy to Drive Adult Cardiac Regeneration
The article explores a novel therapeutic strategy for cardiac regeneration by targeting miRNA‐1a and miRNA‐15b. Combinatorial inhibition of miR‐1a and miR‐15b enhances cardiomyocyte proliferation, improves heart function, and reduces fibrosis in myocardial infarction models.
Ting Yuan+16 more
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G-Quadruplex-Based Splice Switching as a Therapeutic Approach in Duchenne Muscular Dystrophy. [PDF]
Iwase R+4 more
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PANEs are specifically designed to enhance the brain delivery efficiency of circDYM compared to commercialized lipid nanoparticles (LNPs). Intranasal administration of PANE‐circDYM effectively alleviated LPS‐induced depressive‐like behaviors by mitigating neuroinflammation and promoting neuroplasticity.
Feng Gao+11 more
wiley +1 more source
The splicing factor PTBP3 promotes COX11 exon skipping, allowing gastric cancer organoids to evade cuproptosis. Antisense oligonucleotide drugs targeting PTBP3‐mediated COX11 alternative splicing, in combination with copper ionophores, promote cuproptosis in organoids, thereby providing a therapeutic approach for gastric cancer peritoneal metastasis ...
Yajing Zhou+11 more
wiley +1 more source