In partnership with patient advocacy groups and industrial collaborators, we have pledged to help democratize access to physiologically relevant human models of neurodegenerative disease. The Early Access axoCells™ derived from patient-derived iPSCs are widely available to the global scientific community.
axoCells™ human iPSC-derived neural stem cells (HD, CAG125), female donor, aged 7, ≥1.5 million cells
Features
- Derived from human iPSCs from a female donor, aged 7, with severe early-onset Huntington’s disease
- Originated from the CENSOi019-B line (HTT: 14/125 CAG, now CAG143), demonstrating somatic instability, a key modifier of disease onset and progression, and CAG expansion in vitro
- Express the typical markers of neural stem cells (PAX6, FOXG1 and Nestin) and mature striatal neuron markers (DARPP32, CTIP2, GABA)
- Can be matured into high-quality striatal neurons and cortical excitatory neurons
- TempO-Seq™ data available on request
Specifications
Number of cells: ≥1.5 million
Donor: female, aged 7
Genetics: Huntington’s, CAG125 (now CAG143)
Price:
$1,550.00
Description
Axol ax0219 iPSC-derived neural stem cells from a juvenile Huntington’s disease (HD) patient provide a physiologically relevant human model for investigating neurodegenerative mechanisms and accelerating therapeutic discovery. These cells are derived from the CENSOi019-B iPSC line, originating from a donor carrying HTT CAG14/CAG125 repeats, characteristic of early-onset HD.
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Maturation potential: can be efficiently matured into striatal neurons or cortical neurons with optimized media and supplements
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Identity: express key striatal, cortical, and neuronal markers, including DARPP32, CTIP2, MAP2, TUJ1, and GABA
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Research applications: striatal neurons show CAG repeat instability driving Huntington’s disease progression, neuronal dysfunction, and degeneration. Used routinely for projects with endpoints including trinucleotide instability, neurite outgrowth, spontaneous firing, mitochondrial function, and protein aggregation.
ax0219 and its origin line have become a vital resource for HD research, providing an in vitro model for early-onset HD, which is often more severe and less understood than adult-onset disease.
User Guides
Early Access products are derived from patient or unaffected donor iPSC lines. These individual lines may exhibit different characteristics in culture due to the unique genetic background and disease risk factors. Our User Guides provide detailed protocols, however we recommend using standard optimization techniques and monitoring cells carefully to take into account the disease phenotype and intrinsic differences in iPSC-derived cells.
If you would like to discuss the protocols, disease models, or suitable endpoint assays in more detail, please reach out to our scientific team.
The CENSOi019-B line provides critical insights into Huntington’s disease progression because it demonstrates CAG repeat instability, a key driver of neuronal dysfunction and degeneration. Data from this line, including CAG expansion analysis showing one repeat every 5 passages, and Whole-Genome Sequencing revealing loss of CAA interruption and disease-modifying SNPs/INDELs, enable researchers to study mechanisms that influence somatic instability, age of onset, and HD severity. When differentiated into striatal neurons, ax0219 supports functional assays such as neurite outgrowth, spontaneous firing, and trinucleotide instability, making it an essential model for developing targeted therapies and understanding early-onset HD, which is often more severe and less understood than adult-onset disease.
CENSOi019-B HD iPSC line exhibits 143 CAG repeats after banking and exhibits repeat expansion.
Phenotypic characterization: morphology
iPSC-derived striatal neurons derived from an unaffected donor (ax0004) cluster after day 25, showing large cell clusters by day 30, while HD striatal neurons (ax0219) derived from CENSOi019-B remain very neuronal throughout the maturation period. These phenotypic differences have been observed repeatedly in these lines.
Phenotypic characterization: immunocytochemistry
HD striatal neurons (ax0219) express CTIP2, DARPP32 and GABA, markers indicative of mature striatal neurons in addition to neuronal markers like TUJ1 and MAP2.
Striatal neurons derived from the CENSOi019-B line (ax0219) longitudinal studies
ax0219 (CENSOi019-B) and ax0004 (Control) were matured to striatal neurons and cultured for 100 days (the control line did not survive beyond 60 days). Samples were taken throughout the maturation process to assess CAG expansion.
Morphology and neurite outgrowth: CENSOi019-B HD line vs unaffected donor
Morphology of HD striatal neurons and control striatal neurons at Day 20 recorded on IncuCyte®. The controls form many more large clusters and this becomes more prominent as they mature over time which is why it becomes more inaccurate as shown on the graphs.
characterization of the hyperexcitability phenotype in iPSC striatal neurons
Representative sodium spike traces from one electrode of control striatal neurons and HD striatal neurons from the HD line at Day 25 of differentiation show a trend toward increased activity in the HD line.
The HD line displays more frequent spikes, indicating greater neuronal activity. Since striatal neurons are inhibitory by nature, minimal activity is expected in neurons from unaffected donor backgrounds.
The HD line shows activity across most electrodes, whereas the control line exhibits activity in only one electrode, further supporting a trend toward hyperexcitability in the HD model.
Applications
Our axoCellsTM striatal neuron progenitors are suitable for investigating Huntington’s disease and for screening candidate therapies for this disease.
Healthy control lines are also available and can be used with high–content imaging, MEA, microfluidic and organ-on-chip (OOC) devices to measure structural and functional responses in neuronal models.
Quality
We maintain or exceed industry-level quality with our ISO:9001-accredited production facility, guided by our rigorous quality control procedures and decades of scientific experience.
All cells come with a full Certificate of Analysis and are officially certified by HPSCreg®, to ensure ethical and biological conformity for your peace of mind.
Licenses & Consent
Axol has obtained all relevant licenses for reprogramming donor samples into iPSCs and differentiating these into striatal neurons for commercial use.
Patient samples used to create these neurons have been ethically sourced and consented for research and commercial use. All cells come with a full Certificate of Analysis and are officially certified by HPSCreg®, to ensure ethical and biological conformity for your peace of mind.
Publications
Glaser, T., Shimojo, H., Ribeiro, D.E. et al. ATP and spontaneous calcium oscillations control neural stem cell fate determination in Huntington’s disease: a novel approach for cell clock research. Mol Psychiatry 26, 2633–2650 (2021). https://doi.org/10.1038/s41380-020-0717-5
Mathkar, Pranav P. et al. ‘Characterization of Neurodevelopmental Abnormalities in iPSC-Derived Striatal Cultures from Patients with Huntington’s Disease’. Journal of Huntington’s Disease, vol. 8, no. 3, pp. 257-269, 20191 Jan. 2019 : 257 – 269. DOI: 10.3233/JHD-180333
Bunting, E. L., Donaldson, J., Cumming, S. A., et al. Antisense oligonucleotide–mediated MSH3 suppression reduces somatic CAG repeat expansion in Huntington’s disease iPSC–derived striatal neurons. Sci. Transl. Med. 17, eaadn4600 (2025). https://doi.org/10.1126/scitranslmed.adn4600
Media and Reagents
Using our kit of specially formulated striatal neuron media ax0333, growth factors BDNF ax139800 and GDNF ax139855, and SureBond–XF coating, mature striatal neurons can be generated in 31 days. We also supply all reagents as standalone items.
