Journal: Signal Transduction and Targeted Therapy

Article Title: Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines

doi: 10.1038/s41392-024-02112-8

Figure Lengend Snippet: Clinical trials based on ASO therapy

Article Snippet: , APOC-III , Olezarsen (AKCEA-APOCIII-LRx) , Phase 1 Phase 2 Phase 3 Phase 3 Phase 3 Phase 3 Phase 3 Phase 3 Phase 3 , GalNAc , Subcutaneous injection , Healthy volunteers HTG, ASCVD HTG HTG, CVD HTG HTG FCS FCS FCS , NCT05579860 NCT05355402 NCT05681351 NCT05610280 NCT05079919 NCT05552326 NCT05185843 NCT05130450 NCT04568434.

Techniques: Clinical Proteomics, Injection, Modification, Eye Drops, Cream, Mutagenesis

Journal: Journal of Lipid Research

Article Title: Foamy monocytes and atherogenesis in mice with combined hyperlipidemia and effects of antisense knockdown of apoCIII

doi: 10.1016/j.jlr.2025.100763

Figure Lengend Snippet: Increased plasma TGs and atherosclerosis in Ldlr −/− mice with transgenic expression of human apoCIII ( Ldlr −/− apoCIIItg). A: Ldlr −/− ApoCIIItg and Ldlr −/− mice were fed WD for 6 weeks. Plasma total TG and total cholesterol levels (n = 4–9 samples/group). B: TG and cholesterol distributions in lipoprotein fractions fractionated by size-exclusion chromatography. Data are presented as TG and cholesterol levels and percentage in each lipoprotein fraction (n = 3–4 samples/group). C: Representative photographs and quantitative analysis of en face Oil Red O staining for atherosclerotic lesions in whole aorta and aortic sinus in mice (n = 6–8 mice/group). D: Staining for CD11c and human apoCIII (hApoCIII) in aortic sinus lesions in mice. ∗ P < 0.05 and ∗∗∗ P < 0.001 compared to Ldlr −/− group.

Article Snippet: At the end of the treatment period, monocyte lipids were quantified using commercial kits (see below), and plasma apoCIII levels were measured using a mouse apoCIII ELISA kit (Avantor, Inc) following the manufacturer’s instructions.

Techniques: Clinical Proteomics, Transgenic Assay, Expressing, Size-exclusion Chromatography, Staining

Journal: Journal of Lipid Research

Article Title: Foamy monocytes and atherogenesis in mice with combined hyperlipidemia and effects of antisense knockdown of apoCIII

doi: 10.1016/j.jlr.2025.100763

Figure Lengend Snippet: Effects of apoCIII ASO treatment on plasma lipids, monocyte phenotypes, and atherosclerosis in Ldlr −/− apoCIIItg mice. Ldlr −/− ApoCIIItg mice fed WD were treated with a GalNac-conjugated ASO against human apoCIII or a GalNac-conjugated CO (and Ldlr −/− mice were treated with CO) at 10 mg/kg body weight weekly for 12 weeks. A: Plasma total TG and cholesterol levels (n = 14–19 mice/group from three independent experiments with 4–7 mice/group in each experiment). B: Representative FACS examples showing SSC and CD11c of CD36 + and CD36 – monocytes (treatment for 4 weeks) and quantification of changes in proportions, SSC, and CD11c MFI of CD36 + monocytes with treatment (n = 11–14 mice/group). C: Representative photographs and quantification of en face Oil Red O staining of whole aorta. D: Representative photographs and quantification of Oil Red O staining in aortic sinus. E: Representative photographs and quantification of CD11c staining in aortic sinus lesions. Data are shown as mean ± SEM. ∗ P < 0.05, ∗∗ P < 0.01, ∗∗∗ P < 0.001 compared to Ldlr −/− mice with CO (A and B); ## P < 0.01, ### P < 0.001 compared to Ldlr −/− ApoCIIItg mice with CO (A and B).

Article Snippet: At the end of the treatment period, monocyte lipids were quantified using commercial kits (see below), and plasma apoCIII levels were measured using a mouse apoCIII ELISA kit (Avantor, Inc) following the manufacturer’s instructions.

Techniques: Clinical Proteomics, Staining

Journal: International Journal of Molecular Sciences

Article Title: Novel RNA-Based Therapies in the Management of Dyslipidemias

doi: 10.3390/ijms26031026

Figure Lengend Snippet: Novel RNA-based therapies in the management of dyslipidemias.

Article Snippet: , , Olezarsen (ISIS 678354, AKCEA-APOCIII-LRx).

Techniques:

Journal: International Journal of Molecular Sciences

Article Title: Novel RNA-Based Therapies in the Management of Dyslipidemias

doi: 10.3390/ijms26031026

Figure Lengend Snippet: Clinical trials—novel RNA-based therapies in the management of dyslipidemias.

Article Snippet: , , Olezarsen (ISIS 678354, AKCEA-APOCIII-LRx).

Techniques: Injection, Clinical Proteomics

Journal: RSC Medicinal Chemistry

Article Title: Hepatocyte targeting via the asialoglycoprotein receptor

doi: 10.1039/d4md00652f

Figure Lengend Snippet: GalNAc–ASO conjugates on the market and in clinical development

Article Snippet: (IONIS-APOCIII-L Rx , AKCEA-APOCIII-L Rx , Ionis) is designed to inhibit the production of apoC-III to treat hypertriglyceridemia.

Techniques: Infection, Coagulation