Antisense Oligonucleotides
Antisense Oligonucleotides (ASOs) usually refer to short, synthetic, single-stranded DNA or RNA (13-30 nucleotides). Following binding to the targeted mRNA or pre-mRNA, ASOs modulates RNA function by several different mechanisms.
1. ASOs can form an RNA–DNA hybrid that becomes a substrate for RNase H, resulting in target mRNA degradation.
2. ASOs can modulate gene expression via steric block of the ribosomal machinery, which can lead to reduced expression, modulation of splicing and/or restoration of a functional protein.
3. Binding of ASOs to pre-mRNA can alter splicing factor recruitment and regulate splicing events.
The first in vivo applications of ASOs showed limited clinical potential because of the high susceptibility of ASOs with an unmodified phosphoribose backbone to rapid degradation by endonucleases and exonucleases. The modifications in backbone, and sugar molecules give ASOs more affinity and stability. Phosphorodiamidate morpholino oligomers (PMO) are very resistant to nuclease and protease degradation and are mostly used in splicing modulation or translation inhibition. Chemical modifications at 2' position of ribose sugar ring such as 2’-O-methyl (2’-O-me), 2’-Fluoro (2’-F), 2’-O-methoxyethyl (2’-MOE) allow oligonucleotide to adopt RNA like C3’-endo sugar pucker, making it thermally stable.
Cy3 labled Diranersen sodium is a Cy3 labled Diranersen sodium.
- Formula: C177H211N63Na15O89P15S15
- Molecular Weight: 5935.36
Frenlosirsen sodium (ION-935918 sodium; ION251 sodium) is an antisense oligonucleotide and also an IRF4 inhibitor. Frenlosirsen sodium inhibits the survival of malignant plasma cells, reduces the burden of myeloma progenitor cells and prevents disease recurrence. Frenlosirsen sodium is applicable to myeloma-related research.
- Formula: C238H337N61O118P16S15
- Molecular Weight: 6917.10
Edutirsen, an antisense oligonucleotide, is a 1-acylglycerol-3-phosphate O-acyltransferase (PNPLA3) synthesis reducer.
- Molecular Weight: 7203.174 (free acid)
IONIS-FGFR4Rx (ISIS 463588) sodium is an antisense inhibitor of fibroblast growth factor receptor 4 (FGFR4), which is promising for research of renal diseases.
- Molecular Weight: 7309.19
IONIS-GCCRRx (ISIS 426115), a glucocorticoid receptor antagonist, is a 2'-O-methoxyethyl (2'-MOE) antisense oligonucleotide (ASO).
- Molecular Weight: 5355.77
AZD8701 (IONIS-1063734) sodium is an antisense oligonucleotide targeting FOXP3 in regulatory T cells (Tregs). AZD8701 sodium can relieve immunosuppression in cancer.
- Formula: C185H233N73O106P18S6
- Molecular Weight: 5925.20
- Formula: C295H410N78Na19O146P19S17
- Molecular Weight: 8537.6 (free acid)
RG-101 sodium is a hepatocyte targeted N-acetylgalactosamine conjugated oligonucleotide that antagonises miR-122. miR-122 is an important host factor for hepatitis C virus (HCV) replication.
- Molecular Weight: 8537.60
RG-101 is a hepatocyte targeted N-acetylgalactosamine conjugated oligonucleotide that antagonises miR-122. miR-122 is an important host factor for hepatitis C virus (HCV) replication.
- Molecular Weight: 5462.34 (free acid)
IONIS-DNM2-2.5Rx sodium is an antisense agent targeting dynamin 2, and has the potential for the research of centronuclear myopathy (CNM).
- Formula: C238H319N67Na16O113P16S15
- Molecular Weight: 6919.13 (free acid)
Bezeparsen sodium is a PCSK9 synthesis inhibitor.
- Formula: C296H434N82O156P20S13Na20
- Molecular Weight: 8673.4 (free acid)
Fesomersen (sodium) is an antisense oligonucleotide designed to inhibit the production of Factor XI.
- Formula: C296H435N83Na20O151P20S15
- Molecular Weight: 8672.6 (free acid)
Donidalorsen (ISIS-721744; IONIS-PKK-LRX) sodium is an antisense oligonucleotide targeting prekallikrein (PKK). Donidalorsen sodium inhibits kallikrein activity and reduces the production of Bradykinin (HY-P0206) by specifically binding to and degrading PKK mRNA in the liver. Donidalorsen sodium can be used in the research of hereditary angioedema.
- Formula: C223H284N74O115P22S22
- Molecular Weight: 7228.00
Tilsotolimod (IMO-2125) is a TLR9 agonist. Tilsotolimod activates the TLR9 signaling pathway, triggers downstream pro-inflammatory and immunostimulatory pathways, enhances the uptake and killing of cancer cells, and induces adaptive immune responses. Tilsotolimod is applicable to research related to melanoma and colorectal cancer.
- Molecular Weight: 7962.60
FITC-labeled Drisapersen (sodium) is Drisapersen labeled with FITC. Drisapersen, a antisense oligonucleotide, induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthesis of partially functional dystrophin in Duchenne muscular dystrophy (DMD) patients with amenable mutations.
- Molecular Weight: 7654.00
FITC-labeled Tominersen (sodium) is the Tominersen labeled with FITC. Tominersen (RG6042) is a second-generation 2′-O-(2-methoxyethyl) antisense oligonucleotide that targets huntingtin protein (HTT) mRNA and potently suppresses HTT production. Tominersen improves survival and reduces brain atrophy in mice. Tominersen can be used for the research of Huntington’s disease (HD).
- Molecular Weight: 4689.85
SPC5001 is a locked nucleic acid (LNA)-modifed antisense oligonucleotide (ASO) complementary to human PCSK9 (proprotein convertase subtilisin/kexin type 9) mRNA. SPC5001 can be used for the research of hypercholesterolemia. SPC5001 sequence: 5′-TGmCTACAAAACmCmCA-3′.
- Molecular Weight: 6370.15
Alicaforsen is an oligonucleotide and immunostimulant targeting human ICAM-1 mRNA. Alicaforsen hybridizes to specific sites to reduce the expression level of ICAM-1. Alicaforsen is applicable to relevant research on psoriasis, rheumatoid arthritis and inflammatory bowel disease.
- Molecular Weight: 7157.00
Apatorsen is a 2'-methoxyethyl-modified antisense oligonucleotide and also a Hsp27 inhibitor. Apatorsen reduces Hsp27 mRNA and protein levels, impairs stress-induced cytoprotective functions, induces cell apoptosis, inhibits tumor growth and prevents metastasis. Apatorsen is applicable to research related to non-small cell lung cancer, castration-resistant prostate cancer, breast cancer, ovarian cancer and bladder cancer.
- Molecular Weight: 3066.63
Farabursen sodium scrambled negative control is the sequence scrambled negative control of Farabursen sodium.
