Oligonucléotides Antisens (ASOs)

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Attachment Chemistry / Linkers Modi?cations Modi?es pre-mRNA splicing of the SMN2 gene,
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Targets huntingtin protein (HTT) mRNA,
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Mediates RNase H-dependent degradation of SOD1 mRNA,
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mRNA compaction.
Targets exon 45 of dystrophin pre-mRNA,
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Spacers Modi?cations
Inotersen Targets the splicing of exon 53 in the dystrophin gene,
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C3 Spacer C6 Spacer C12 Spacer dSpacer PC-linker (sodium) used for the research of the DMD.
References:
Targets exon 53 of dystrophin pre-mRNA used for
Spacer 18 Spacer 9 HY-132611 Golodirsen ASO [1] Front Bioeng Biotechnol. 2021;9:628137. [2] J Neuromuscul Dis. 2020;7(1):1-13. [3] Chonnam Med J. 2020;56(2):87-93.
the research of Duchenne muscular dystrophy (DMD). [4] Nat Nanotechnol. 2021;16(6):630-643. [5] Nat Rev Neurol. 2018;14(1):9-21. [6] Adv Drug Deliv Rev. 2020;154-155:37-63.
[7] Nat Rev Genet. 2022 May;23(5):265-280. [8] Mol Cancer. 2021;20(1):54. [9] Cells. 2020;9(1):137. Published 2020 Jan 7.
Inhibits miR-17 function?used for the research of
HY-139290 RGLS4326 ASO [10] Adv Drug Deliv Rev. 2018;134:65-78.
Modi?ed Bases autosomal dominant polycystic kidney disease (ADPKD)

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Master of Bioactive Molecules Inhibitors • Screening Libraries • Proteins
SiRNAs are small exogenous double-stranded RNA (dsRNA) (20-25 nucleotides), which triggers the RNA interference Aptamers are short single-stranded DNA or RNA, which can bind to various targets, such as proteins, peptides,
(RNAi) pathway. The short dsRNA unwinds and the sense strand is degraded. Antisense strand forms RNA induced carbohydrates, and other molecules, by virtue of their tertiary structures, rather than their sequences. The aptamers
Oligonucleotides silencing complex (RISC) with various protein components. The antisense strand retained in RISC is speci?cally have a high af?nity to target proteins similar to antibodies. Compared to antibodies, however, aptamers are small in size Synthesis Services
complementary to the target gene mRNA. Meanwhile, RISC has nuclease activity, which can cut and degrade the target and have additional advantages, including improved transport into the cells and lower cost [1][4][10].
Conventional drug strategy relies on the ability of small molecule drugs to target active sites of proteins to inhibit or alter gene mRNA, and inhibit the expression of target gene. Incomplete complementarity results in mRNA translation inhibition [8]. MCE owns a professional team and state-of-the-art facilities in the production and analysis of oligonucleotides. MCE can
their function. However, only 10–14% of proteins have active binding sites that are druggable targets for small molecules,
MicroRNAs (miRNAs) are endogenous non-coding RNAs that contain approximately 22 nucleotides, and their primary synthesize oligonucleotides to suit your speci?c needs, carry out related testing procedures (purity, structure and stability
the vast majority of proteins cannot be targeted for small molecules [1]. There is no cure for some rare diseases, for 01
function is to mediate gene silencing. MiRNAs usually bind to 3'-UTR of mRNA. For mammals, the base-pairing is always analysis), and ensure the accuracy and reproducibility of data with high-quality and ef?cient services.
example, the use of small molecule drugs (such as Valproic acid, Albuterol and Riluzol) to treat spinal muscular atrophy Antisense
imperfect, resulting in the suppression of mRNA translation. In contrast, most plant miRNAs bind with near-perfect Oligonucleotides
(SMA) is not much effective [2]. This limitation was addressed in part by the revolution of small nucleic acid drugs.
complementarity to sites within the coding sequence of their targets, and the mRNA of the target gene is sliced and
Antisense oligonucleotide, Nusinersen (Spinraza®) is the only approved therapy for SMA in 2016 [2]. Advantages
degraded. In addition, miRNAs can have multiple targets, because they act through less complementarity [8][9]. 06 02
The utilization of oligonucleotides as drugs is a relatively novel approach as compared to conventional small molecule
CpG ODNs Aptamers
inhibitors. The potential of RNA therapies in precision genetics has raised enthusiasm for similar applications in cancer, Comprehensive modi?cations Reliable quality data Powerful capacity
cardiovascular diseases, and rare diseases therapies. The recent FDA approvals of Givosiran, Lumasiran and Viltolarsen Nucleus
have ushered the wave of RNAi or RNA-based therapies into the mainstream of drug development. RNA polymerase Professional technical support
pri-miRNA
Oligonucleotides are composed of nucleotides with specially designed sequences. Most of the oligonucleotides hybridize Oligonucleotides
DNA
with the target gene mRNA or pre-mRNA through complementary base pairing, and can theoretically selectively regulate Drosha
any target gene and protein expression, including many “undruggable” targets. This means that oligonucleotides have the mRNA
Cytoplasm
potential to be used for many rare diseases whose pathogenesis is still unclear. Oligonucleotides also have additional 05 03 Related Synthesis Services
advantages, including relatively simple production and preparation technology, short development cycles, and miRNAs Cap Analogs
pre-miRNA
long-lasting effect. • siRNAs: single gene siRNA set and siRNA libraries.
• miRNAs: miRNA libraries (miRNA mimics or miRNA inhibitors).
Currently, common oligonucleotides are antisense oligonucleotides (ASOs), siRNA (small interfering RNA), microRNA
and aptamers[1][3]. 04 • Oligos: various modi?cations (Locked Nucleic Acid (LNA), Phosphorothioate, 2’-OMe, 2’-MOE, 2'-Fluoro, CY3, CY5, FAM,
Exportin-5
shRNA siRNAs etc.)
ASOs usually refer to short, synthetic, single-stranded DNA or RNA (13-30 nucleotides) [4]. Following binding to the
dsRNA • Oligonucleotide-conjugates?PEGs, lipids, small molecule compounds, polypeptides, etc.
targeted mRNA or pre-mRNA, ASOs modulate RNA function by several different mechanisms [5][6][7]. pre-miRNA • Preparation of lipid nanoparticles (LNPs).
Dicer
1. ASOs can form an RNA–DNA hybrid that becomes a substrate for RNase H, resulting in target mRNA degradation. Dicer 01
2. ASOs can modulate gene expression via steric blocking of the ribosomal machinery, which can lead to reduced AGO
Guide strand Cationic Lipids
expression, modulation of splicing and/or restoration of a functional protein. Passenger strand RISC
3. Binding of ASOs to pre-mRNA can alter splicing factor recruitment and regulate splicing events. siRNA RISC Loading 01
Loading miRNA 02 MCE offers more than 100 modi?cations (only some commonly used modi?cations are listed).
Nucleosides
Pegylated
and
ASO : ~6 kDa
their Analogs Lipids
Passenger strand is cleaved Fluorophores
(after loadin g)
siRISC miRISC Nucleosides Alexa Fluor 488 AMCA AquaPhluor 593 Atto 425 Atto 590
02 and Lipids
RNAase H1 AGO AGO 03 BODIPY FL Cy3 Cy5 Cy5.5 Cy7
Ribosome
Splice Nucleotides Nucleotides
Phospholipids
repressor and
mRNA 5’ G their Analogs FAM HEX JOE 02 NED Paci?c Blue
A A A A A A 3’
Quasar 570 Quasar 670 ROX TAMRA TET
Target mRNA is sliced
RNAase H1-mediated & degraded, translation Translation suppression 03 04
mRNA silencing repression Texas red VIC Yakima Yellow
Translation arrest by Splicing modulation by Nucleoside Cholesterol
blocking the ribosome splice repressor occlusion Phosphoram
-idites
Figure 1. The expanding universe of therapeutic RNA payloads [7]. Figure 2. Schematic illustrations for siRNA/miRNA biogenesis and, the mRNA inhibition via siRNA and miRNA-mediated mechanisms [8].