Mivelsiran sodium
Based on 1 Customer Validation
Mivelsiran (ALN-APP) sodium is an siRNA targeting the β-amyloid precursor protein (APP). Mivelsiran sodium induces sequence-specific degradation of APP mRNA via RNA interference, thereby reducing the levels of APP protein and downstream amyloid cleavage products. Mivelsiran sodium reduces β-amyloid protein levels in the brain, alleviates neuroinflammation, and attenuates microglial proliferation. Mivelsiran sodium can be used for research on early-onset Alzheimer's disease.
For research use only. We do not sell to patients.
- Purity : 95.46%
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Storage:
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Biological Activity
Description
In Vitro
Mivelsiran sodium does not induce cytokine release or immune activation in ex vivo human whole blood[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only. Further protocols information, click here.
In Vivo
Mivelsiran (60-120 µg; i.c.v.; single dose) sodium suppresses APP expression, reduces neuroinflammation, and normalizes hyperactive behavior in cerebrovascular amyloid Nos2−/− (CVN) Alzheimer's disease mice[1].
Mivelsiran (300 µg; i.c.v.; two doses at 3 and 6 months, single dose at 8 months) sodium reduces amyloid beta levels, neuroinflammatory markers, and abnormal anxiety-like behavior in 5xFAD Alzheimer's disease mice when administered early, while late intervention does not improve behavioral deficits despite reducing pathological markers[1].
MedChemExpress (MCE) has not independently confirmed the accuracy of these methods. They are for reference only.
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Animal Model:Sprague Dawley (male)[1]
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Dosage:0.07 mg; 0.3 mg; 0.9 mg
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Administration:i.t.; single dose; monthly for 5 consecutive months
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Result:Reduced SOD1 mRNA to below 50% in the thoracic spinal cord, cerebellum, and frontal cortex at day 28 for all single doses.
Reduced SOD1 mRNA to 25% of control levels across thoracic spinal cord, cerebellum, and frontal cortex with the 0.9 mg dose.
Maintained silencing exceeding 50% for over three months in most CNS regions (spinal cord segments, cerebellum, frontal cortex, temporal cortex, hippocampus) with the 0.9 mg dose.
Maintained silencing above 50% for five months in the spinal cord and frontal cortex with the 0.9 mg dose.
Produced positive silencing effects over five months with the 0.3 mg monthly dosing regimen.
Showed cranial distribution via cerebrospinal fluid, reaching the cortex, hippocampus, olfactory bulb, and brainstem, with uptake observed in neurons, astrocytes, and microglia.
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Animal Model:Cynomolgus Monkey[1]
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Dosage:45 mg; 60 mg
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Administration:i.t.; single dose
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Result:Achieved 70-80% knockdown of target mRNA across multiple CNS regions with the 60 mg dose.
Reached maximal silencing within one week, sustained above 75% for ~2.5 months, declined to 50% by 4.5 months, and returned near baseline by 9 months with the 60 mg dose.
Reduced cerebrospinal fluid levels of soluble APPα (sAPPα) and soluble APPβ (sAPPβ) to below 25% for up to two months, and below 50% for five months with both doses.
Showed no treatment-related microscopic neuropathology in the brain, spinal cord, or dorsal root ganglia.
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Animal Model:Homozygous Tg-hAPPSwDl/mNos2−/− (CVN) (6-12 months old)[1]
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Dosage:60 µg; 120 µg
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Administration:i.c.v.; single dose
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Result:Reduced APP expression (mRNA and peptide levels) at 30 days post-administration with the 60 µg dose.
Suppressed APP mRNA in the ventral cortex for 60 days with the 120 µg dose, with effects observed in other brain regions over extended time points.
Showed a non-statistically significant reduction in Aβ40 immunostaining in the cortex and hippocampus.
Showed a statistically significant reduction in Iba1 immunostaining (microgliosis) in the cortex and hippocampus compared to untreated controls.
Exhibited reduced distance traveled and rearing frequency in the open field test, indicating mitigated hyperactivity.
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Animal Model:5xFAD (transgenic, expressing five familial Alzheimer's disease mutations)[1]
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Dosage:300 µg
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Administration:i.c.v.; two doses at 3 and 6 months; single dose at 8 months
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Result:Significantly decreased cortical Aβ40 and Aβ42 levels with early intervention (two doses at 3 and 6 months).
Reduced plasma neurofilament light chain (NfL) and glial fibrillary acidic protein (GFAP) levels with early intervention (two doses at 3 and 6 months).
Produced a dose-dependent decrease in time spent in the open arms of the elevated plus maze, with the robust early intervention group exhibiting behavior comparable to healthy controls at 8 and 12 months.
Reduced Aβ40, Aβ42, NfL, and GFAP levels with late intervention (single dose at 8 months).
Did not normalize elevated plus maze behavior with late intervention (single dose at 8 months).
Chemical Information
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Appearance Solid
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Color White to off-white
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SMILES
[Mivelsiran (sodium)]
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Synonyms
ALN-APP sodium
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Shipping
Room temperature in continental US; may vary elsewhere.
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Storage
-20°C, sealed storage, away from moisture
* In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)
Protocols
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RNA extraction experimental
By lysing cells, releasing RNA, and removing impurities such as proteins and DNA, high-purity RNA products are finally obtained. The commonly used traditional method is the guanidine isothiocyanate/phenol/chloroform method (Trizol), which is suitable for a variety of animal materials including animal tissues, microorganisms, cultured cells, etc., and most plant materials.
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RNA interference technology
RNA interference (RNAi) is a cellular mechanism that inhibits gene expression by suppressing gene transcription or activating RNA degradation. This mechanism was discovered in plants in 1998 by Andrew Fire and Craig Mello. Today, this phenomenon can be observed in almost all eukaryotes, including protozoa, flies, nematodes, insects, parasites, and mammals.
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Research Protocol for Neurological Diseases
PINK1/Parkin-mediated mitophagy pathway is a mitochondrial quality-control signaling axis in which mitochondrial depolarization stabilizes PINK1 on damaged mitochondria, activates Parkin recruitment and E3 ubiquitin ligase activity, promotes ubiquitination of outer mitochondrial membrane proteins, recruits selective autophagy adaptors, and drives lysosomal degradation of damaged mitochondria. In neurological disease research, this pathway is experimentally important because neurons, especially dopaminergic neurons, are highly dependent on mitochondrial integrity, and defective mitochondrial turnover can lead to mitochondrial dysfunction, oxidative stress, impaired neuronal survival, α-synuclein accumulation, and neuroinflammatory damage-associated signals. The genetic disease link is strongest in Parkinson’s disease because mutations in PRKN/parkin cause autosomal recessive juvenile parkinsonism, mutations in PINK1 cause hereditary early-onset Parkinson’s disease, and Drosophila studie
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Amyloid: Congo Red Amyloid Staining
Congo red amyloid staining is a histochemical method used to detect extracellular amyloid deposits in tissue sections based on the affinity of Congo red dye for β-pleated sheet-rich protein aggregates. When bound to amyloid, Congo red produces characteristic apple-green birefringence under polarized light microscopy, which is widely regarded as a diagnostic feature of amyloid deposition in histopathology. The diagnostic principle relies on the combination of dye binding (congophilia) and optical anisotropy under polarized illumination, which distinguishes amyloid from most non-amyloid eosinophilic extracellular deposits in routine histological evaluation. Amyloid identification by Congo red staining remains a cornerstone in diagnostic pathology despite the availability of adjunct methods such as immunohistochemistry and mass spectrometry, particularly because of its ability to localize deposits directly within tissue architecture. The specificity of Congo red-positive deposits is incre
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Cell Viability Determination by MTT Colorimetric Assay
The following protocol uses the MTT colorimetric assay as a classic literature-established method for assessing cell viability/metabolic activity in cultured mammalian cells. MTT[3-(4,5-dimethylthiazol-2-yl)-2,5-diphenyltetrazolium bromide] is reduced by metabolically active cells to a colored formazan product; the amount of formazan is quantified spectrophotometrically and provides an indirect measure of metabolically active viable cells. Importantly, MTT reduction reflects cellular oxidoreductase/metabolic activity rather than an absolute direct count of living cells, so changes in cellular metabolism can alter the signal independently of cell number.
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Alzheimer’s Disease Modeling
Alzheimer’s Disease (AD) is a neurodegenerative disorder characterized by a progressive decline in cognitive functions and loss of specific types of neurons and synapses. Alzheimer's symptoms can be simulated in mice by injecting drugs (such as Aβ) or genetically modified.
Purity & Documentation
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Data Sheet (279 KB)
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SDS (252 KB)
- English - EN (252 KB)
- Français - FR (252 KB)
- Deutsch - DE (252 KB)
- Norwegian - NO (252 KB)
- Español - ES (252 KB)
- Swedish - SV (252 KB)
- Italian - IT (252 KB)
- Korean - KR (252 KB)
- Portuguese - PT (252 KB)
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Handling Instructions (2242 KB)
References
Calculators
Concentration (start) × Volume (start) = Concentration (final) × Volume (final)