Antisense Oligonucleotides (ASOs)
Creative Biolabs supports antisense oligonucleotides (ASOs) development and manufacturing solutions with the integrated platform of a leading CRO company. Not only do we have ready-to-use ASO products for you to choose from, but we are also committed to customizing products for you through our services.
Product Overview
ASOs are chemically modified short-chain nucleic acids that range in length from 15 to 25 nucleotides. It is dependent on RNase H to function and could specifically degrade RNA in the cytoplasm and nucleus. ASOs mostly treat diseases by silencing mRNA, inhibiting ribosome synthesis protein, and regulating RNA splicing. The mechanism of action of ASOs, as well as improvements in clinical trial design, have played major roles in accelerating clinical transformation based on ASO strategies, particularly for the treatment of multiple neurological diseases.
Fig. 1 The mechanisms of ASOs.
Case Study
Microglia express apolipoprotein E (APOE) and triggering receptor expressed on myeloid cells 2 (TREM2), the two most potent risk factors for Alzheimer's disease. Neurodegeneration and brain homeostasis are two processes in which microglia are essential. The lack of cross-species conservation in the sequence, structure, and function of a number of microglial proteins hinders the advancement of methods for regulating the expression of particular microglial genes. Using ASOs to regulate APOE and TREM2 expression is one way to target these genes.
After four weeks, transplanted human microglia that have been exposed to ASOs targeting APOE and TREM2 exhibit reduced responses to amyloid-β plaques due to rapid transcriptional changes. ASOs that target human microglia in an AD model have the ability to alter the transcriptional patterns and in vivo responses of these cells to amyloid plaques.
Fig. 2 Representative images show activated human microglia targeting X-34-positive amyloid-β fibrils 4 weeks after APOE ASO and TREM2 ASO treatment.1,2
Advantages of ASOs
- ASOs can be produced rapidly, within a week. The only information required is the sequence of the mRNA.
- Inhibiting mRNA expression can lead to faster and more durable clinical responses than traditional drug targeting of proteins.
- ASOs are known to accumulate in particular tissues and organs, including adipocytes, the liver, spleen, kidney, and bone marrow. There are several ways to administer them.
Creative Biolabs, one of the world's premier biotechnology companies, boasts modern equipment and an experienced workforce. Creative Biolabs' scientists can supply you with customized ASO synthesis services. We also offer a selection of ready-to-use ASO products for various disease research; please contact us to discuss your unique project or learn more about our ASO products.
Reference
- Vandermeulen, Lina, et al. "Regulation of human microglial gene expression and function via RNAase-H active antisense oligonucleotides in vivo in Alzheimer's disease." Molecular Neurodegeneration 19.1 (2024): 37. Distributed under Open Access license CC BY 4.0, without modification.
Target





Human Natural ABCA1 antisense sequence

- Applications:
- Modulates IL-6 expression to investigate its role in neuroinflammation, blood-brain barrier permeability, and neuronal survival in multiple sclerosis (MS), AD, and stroke. Suitable for in vitro (glial cell cultures) and in vivo (MS/EAE mouse models) studies to validate IL-6 as an anti-inflammatory target.

- Applications:
- Inhibits NLRP3 inflammasome activation to study its role in neuroinflammation, microglial priming, and neurodegeneration in AD, PD, and ALS. Enables research on NLRP3-mediated cytokine release and neurotoxicity. Ideal for in vitro (inflammasome assays) and in vivo (neuroinflammation models).

- Applications:
- Reduces TDP-43 expression to investigate its role in RNA processing, protein aggregation, and motor neuron degeneration in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Suitable for in vitro (motor neuron cultures) and in vivo (ALS mouse models) studies.

- Applications:
- Inhibits PINK1 expression to study its role in mitochondrial quality control, mitophagy, and neuronal survival in PD. Facilitates research on PINK1-mediated pathways for neuroprotection. Suitable for in vitro (mitochondrial stress assays) and in vivo (PINK1 mutant mouse models).

- Applications:
- Modulates DJ-1 expression to investigate its role in oxidative stress response, mitochondrial function, and dopaminergic neuron survival in PD. Enables studies on DJ-1's protective mechanisms against neurodegeneration. Ideal for in vitro (neuroblastoma cell lines) and in vivo (DJ-1 knockout mouse models).

- Applications:
- Targets SNCA mRNA to decrease α-synuclein aggregation in PD and dementia with Lewy bodies (DLB). Facilitates studies on α-synuclein's role in synaptic toxicity, mitochondrial dysfunction, and neuroinflammation. Suitable for in vitro (dopaminergic neuron cultures) and in vivo (PD mouse models) research.

- Applications:
- Reduces LRRK2 expression to study its role in neuronal autophagy, mitochondrial dysfunction, and dopaminergic neurodegeneration in Parkinson's disease (PD). Enables preclinical evaluation of LRRK2 inhibition for neuroprotection. Optimized for in vivo PD models (e.g., α-synuclein transgenic mice).

- Applications:
- Inhibits TREM2 expression to investigate its role in microglial activation, amyloid phagocytosis, and neuroinflammation in AD. Suitable for in vitro (microglia cultures) and in vivo (AD mouse models) studies to validate TREM2 as a therapeutic target for modulating innate immune responses in the brain.

- Applications:
- Modulates ApoE expression to study its role in lipid transport, amyloid clearance, and neuroinflammation in AD. Enables exploration of ApoE isoform-specific effects (e.g., ApoE4 vs. ApoE3) on tau pathology and neuronal survival. Ideal for in vitro (microglial-neuronal co-cultures) and in vivo (ApoE knockout mouse models) research.

- Applications:
- Inhibits BACE1 expression to reduce Aβ peptide generation in AD research. Facilitates studies on BACE1-mediated synaptic dysfunction, neuroinflammation, and blood-brain barrier integrity. Optimized for preclinical models evaluating BACE1-targeted therapies, including in vivo assessments of cognitive function in AD mice.

- Applications:
- Specifically targets APP mRNA to reduce amyloid-β (Aβ) production in Alzheimer's disease (AD) research. Enables investigation of APP's role in neuronal plasticity, Aβ aggregation, and tau phosphorylation. Suitable for in vitro (primary neuronal cultures) and in vivo (AD transgenic mouse models) studies to validate APP as a therapeutic target for Aβ-related neurodegeneration.

- Applications:
- Regulates lipid metabolism with potential CNS effects. In neuroscience, explores ANGPTL3's role in blood-brain barrier lipid transport, neuroinflammation, and neurodegeneration (e.g., Alzheimer's). Optimized for in vivo models of hyperlipidemia and AD.

- Applications:
- Modulates NRG-1/HER4 signaling in schizophrenia and glioblastoma. In neuroscience, investigates HER4's role in oligodendrocyte maturation, myelination, and neuronal migration. Optimized for in vitro (oligodendrocyte precursors) and in vivo (schizophrenia mouse models).

- Applications:
- Blocks Raf/MEK/ERK pathway activation in neurodegeneration and brain cancer. In neuroscience, evaluates C-Raf's role in neuronal survival, apoptosis, and glioblastoma invasion. Ideal for in vitro (neuronal/glial cultures) and in vivo (glioma/xenograft models).

- Applications:
- Inhibits Ras/MAPK signaling to study neuronal differentiation, axon guidance, and glioblastoma growth. In neuroscience, investigates Ha-Ras's role in neurodevelopmental disorders (e.g., Noonan syndrome) and brain tumorigenesis. Optimized for in vitro (neural stem cells) and in vivo (glioma models).

- Applications:
- Studies insulin resistance in Alzheimer's disease (AD) and type 2 diabetes-related cognitive decline. By targeting IRS-1, explores its role in neuronal insulin signaling, amyloid-beta clearance, and tau phosphorylation. Ideal for in vitro (neuronal cultures) and in vivo (diabetes/AD mouse models).

- Applications:
- Modulates excitatory neurotransmission in epilepsy, stroke, and neurodegeneration. By targeting AMPA/NMDA receptor subunits, investigates synaptic plasticity, excitotoxicity, and seizure propagation. Optimized for in vitro electrophysiology and in vivo seizure models.

- Applications:
- Selectively inhibits PKA-I isoform to study cAMP signaling in learning/memory and mood disorders. In neuroscience, investigates PKA-I's role in long-term potentiation (LTP), CREB phosphorylation, and antidepressant response. Ideal for in vitro (hippocampal slices) and in vivo (behavioral assays).

- Applications:
- Studies endocytosis defects in Charcot-Marie-Tooth neuropathy (CMT) and centronuclear myopathy. In neuroscience, explores DNM2's role in synaptic vesicle recycling, axonal transport, and motor neuron survival. Optimized for in vitro (neuronal cultures) and in vivo (CMT mouse models).

- Applications:
- Modulates immune cell trafficking and neural progenitor migration in multiple sclerosis (MS) and stroke. By targeting CXCL12, investigates its role in lesion repair, remyelination, and post-stroke neurogenesis. Ideal for in vitro (brain endothelial/immune cell co-cultures) and in vivo (EAE/MS models).

- Applications:
- Potent cell-penetrating ASO for targeting "undruggable" RNAs in neurodegenerative diseases. In neuroscience, validates PPMO's efficacy against repeat expansions (e.g., C9ORF72 in ALS/FTD) or toxic transcripts. Optimized for in vitro and in vivo delivery to neurons and glia.

- Applications:
- Corrects splicing defects in USH2A mutations linked to Usher syndrome (deafness and retinitis pigmentosa). In neuroscience, investigates USH2A's role in inner ear hair cell synapses and photoreceptor-neuron interactions. Enables preclinical testing of exon skipping strategies for genetic hearing/vision loss.

- Applications:
- Modulates bradykinin production and inflammation in angioedema and CNS trauma. In neuroscience, explores PKK's role in blood-brain barrier disruption, neuroinflammation, and edema formation following TBI or subarachnoid hemorrhage. Ideal for in vivo models of neurovascular injury.

- Applications:
- Reduces thrombosis risk while minimizing bleeding complications in CNS disorders. In neuroscience, evaluates FXI's role in cerebral microvascular thrombosis, blood-brain barrier integrity, and post-stroke inflammation. Optimized for preclinical models of ischemic stroke and cerebral amyloid angiopathy.

- Applications:
- Modulates cellular response to hypoxia in stroke, traumatic brain injury (TBI), and neurodegeneration. By targeting HIF-1α, investigates angiogenesis, neuronal survival, and metabolic reprogramming in ischemic/hypoxic brain regions. Validates HIF-1α as a therapeutic target for neuroprotection and recovery.

- Applications:
- Regulates hepatic glucagon signaling with potential CNS effects. In neuroscience, explores GCGR's role in brain glucose uptake, ketone body production, and neuroprotection during metabolic stress (e.g., stroke, hypoglycemia). Optimized for liver-brain axis research, suitable for in vivo models of metabolic disorders.

- Applications:
- Modulates insulin/glucagon signaling in neurodegenerative diseases (e.g., Alzheimer's) by targeting PTP-1B, a negative regulator of insulin receptor phosphorylation. Enables investigation of PTP-1B's role in neuronal energy metabolism, amyloid-beta clearance, and synaptic plasticity. Ideal for in vitro (neuronal/glial cultures) and in vivo (diabetes/AD mouse models) studies.

- Applications:
- Our FOXP3-ASO targets Forkhead Box P3 (FOXP3), which is involved in regulatory T - cell function. In neuroscience, it allows researchers to investigate FOXP3's role in neuroimmune regulation, neuroinflammation, and autoimmune neurological diseases, facilitating the development of immunomodulatory therapies.
- iNeuMab™ Mouse Anti-LRP1 Monoclonal Antibody (CBP3363) (Cat#: NAB-0720-Z6479)
- iNeuMab™ Anti-Integrin αvβ8 BBB Shuttle Antibody (NRZP-1222-ZP1218) (Cat#: NRZP-1222-ZP1218)
- iNeuMab™ Anti-Tau Antibody (NRP-0422-P1683) (Cat#: NRP-0422-P1683)
- iNeuMab™ Anti-TNFα BBB Shuttle Antibody (NRZP-1022-ZP4105) (Cat#: NRZP-1022-ZP4105)
- iNeuMab™ Anti-GARP Antibody (NRP-0422-P1639) (Cat#: NRP-0422-P1639)
- iNeuMab™ Anti-GD2 Antibody (NRZP-1222-ZP767) (Cat#: NRZP-1222-ZP767)
- iNeuMab™ Mouse Anti-EFNB2 Monoclonal Antibody (CBP1159) (Cat#: NAB-0720-Z4396)
- iNeuMab™ Anti-Alpha Synuclein Antibody (NRP-0422-P614) (Cat#: NRP-0422-P614)
- Mouse Anti-Human α-Synuclein Phospho (Tyr39) (CBP3706) (Cat#: NAB201250LS)
- iNeuMab™ Anti-CD32b Antibody (NRP-0422-P1803) (Cat#: NRP-0422-P1803)
- Mouse Glioma Cell Line GL261-GFP (Cat#: NCL-2108P04)
- Rat Glioma Cell Line C6 (Cat#: NCL2110P346)
- iNeu™ Human Neural Stem Cell Line (Cat#: NCL200552ZP)
- Human Glial (Oligodendrocytic) Hybrid Cell Line (MO3.13) (Cat#: NCL-2108P34)
- iNeu™ Human Schwann Cell (Cat#: NCL-2103-P63)
- Immortalized Human Cerebral Microvascular Endothelial Cells (Cat#: NCL-2108-P020)
- Mouse Retinal Ganglion Cells (Cat#: NCL2110P145)
- Human Dental Pulp Stem Cells (Cat#: NRZP-1122-ZP113)
- Rat Retinal Muller Cell Line, Immortalized (Cat#: NCL-21P6-192)
- Rat Microglia Cell Line HAPI, Immortalized (Cat#: NCL2110P015)
- Human GFAP ELISA Kit [Colorimetric] (Cat#: NPP2011ZP383)
- Human Tau Aggregation Kit (Cat#: NRP-0322-P2173)
- Human Poly ADP ribose polymerase,PARP Assay Kit (Cat#: NRZP-1122-ZP62)
- Amyloid beta 1-42 Kit (Cat#: NRP-0322-P2170)
- Beta Amyloid (1-42), Aggregation Kit (Cat#: NRZP-0323-ZP200)
- Beta Amyloid (1-40), Aggregation Kit (Cat#: NRZP-0323-ZP199)
- Alpha-Synuclein Aggregation Assay Kit (Cat#: NRZP-1122-ZP37)
- Alpha Synuclein Aggregation Kit (Cat#: NRZP-1122-ZP15)
- VSV-eGFP (Cat#: NTA-2011-ZP20)
- Dextran, NHS Activated (Cat#: NRZP-0722-ZP124)
- AAV2 Full Capsids, Reference Standards (Cat#: NTC2101070CR)
- Human huntingtin (HTT) (NM_002111) ORF clone, Myc-DDK Tagged (Cat#: NEP-0521-R0497)
- App Rat amyloid beta (A4) precursor protein (App)(NM_019288) ORF clone, Untagged (Cat#: NEP-0421-R0053)
- Human superoxide dismutase 3, extracellular (SOD3) (NM_003102) ORF clone, Untagged (Cat#: NEP-0521-R0808)
- Mouse Parkinson disease (autosomal recessive, early onset) 7 (Park7) (NM_020569) clone, Untagged (Cat#: NEP-0621-R0133)
- Mouse SOD1 shRNA Silencing Adenovirus (Cat#: NV-2106-P14)
- Human presenilin 1 (PSEN1), transcript variant 2 (NM_007318) ORF clone, TurboGFP Tagged (Cat#: NEP-0421-R0140)
- Tau Antisense Oligonucleotide (IONIS-MAPTRx) (Cat#: NV-2106-P29)
- ABCA1 Antisense Oligonucleotide (NV-2106-P27) (Cat#: NV-2106-P27)
- Human huntingtin-associated protein 1 (HAP1) transcript variant 2 (NM_177977) ORF clone, Myc-DDK Tagged (Cat#: NEP-0521-R0676)
- Rat Parkinson disease (autosomal recessive, juvenile) 2, parkin (Park2) (NM_020093) ORF clone/lentiviral particle, Myc-DDK Tagged (Cat#: NEP-0621-R0041)
- NeuroBiologics™ Human Cerebrospinal Fluid (Cat#: NRZP-0822-ZP491)
- NeuroBiologics™ Pig Cerebrospinal Fluid (Cat#: NRZP-0822-ZP498)
- NeuroBiologics™ Mouse Cerebrospinal Fluid (Cat#: NRZP-0822-ZP497)
- NeuroBiologics™ Rat Cerebrospinal Fluid (Cat#: NRZP-0822-ZP496)
- NeuroBiologics™ Monkey Cerebrospinal Fluid (Cat#: NRZP-0822-ZP495)
- NeuroPro™ Anti-Erythropoietin BBB Shuttle Protein (Cat#: NRZP-0423-ZP499)
- NeuroPro™ Anti-NAGLU BBB Shuttle Protein (Cat#: NRZP-0423-ZP506)
- NeuroPro™ Anti-IDUA BBB Shuttle Protein (Cat#: NRZP-0423-ZP502)
- NeuroPro™ Anti-EPO BBB Shuttle Protein (Cat#: NRZP-0423-ZP508)
- NeuroPro™ Anti-ASA BBB Shuttle Protein (Cat#: NRZP-0423-ZP504)
- NeuroPro™ Anti-TNFR BBB Shuttle Protein (Cat#: NRZP-0423-ZP510)
- NeuroPro™ Anti-SGSH BBB Shuttle Protein (Cat#: NRZP-0423-ZP505)
- NeuroPro™ Anti-IDS BBB Shuttle Protein (Cat#: NRZP-0423-ZP503)
- NeuroPro™ Anti-PON1 BBB Shuttle Protein (Cat#: NRZP-0423-ZP507)
- NeuroPro™ Anti-idursulfase BBB Shuttle Protein (Cat#: NRZP-0423-ZP497)