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Stem cell treatment & Friedreich ataxia

Checked 11 Nov 2026 · Friedreich ataxia

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Written from the studies on PubMed, searched on 11 Nov 2026. Every number below comes from a study listed under Sources.

The claim

Posts online say that stem cell treatment helps with Friedreich ataxia.

Where research stands

We found 41 published papers on stem cell treatment and Friedreich ataxia. They were published between 2009 and 2026. None of them is a study in people. 8 were studies in animals, and 26 were only in cells in the lab. 3 papers are reviews that sum up other studies.

  • 0In people
  • 8In animals
  • 26Only in the lab

What the studies found

We have not summed up these studies in words yet. The most important papers are listed under Sources.

Verdict

Unproven

Stem cell treatment has been studied for Friedreich ataxia in animals, not yet in people.

Unproven means: it was only tested in animals or cells, or there is too little or mixed data from people. How we rate claims

What this means for you: Results in animals or in cells often do not hold in people. Do not stop or change your medicines because of it. Ask your doctor before you start taking it.

Sources

  1. Human pluripotent stem cell models of Friedreich's ataxia: innovations, considerations, and future perspectives.2026 · Stem Cell Res Ther · Review
  2. Using human pluripotent stem cells to study Friedreich ataxia cardiomyopathy.2016 · Int J Cardiol · Review
  3. Cell and gene therapy for Friedreich ataxia: progress to date.2014 · Hum Gene Ther · Review
  4. Cells heal themselves: gene-edited hematopoietic stem cells restore frataxin deficiency in multiple organs.2026 · J Clin Invest · In animals
  5. Therapeutic activity of a hematopoietic stem cell-delivered cell-penetrating frataxin in Friedreich's ataxia models.2026 · Cell Rep Med · In animals
  6. CRISPR-Cas9 Gene Editing of Hematopoietic Stem Cells from Patients with Friedreich's Ataxia.2020 · Mol Ther Methods Clin Dev · In animals
  7. Nrf2 Induction Re-establishes a Proper Neuronal Differentiation Program in Friedreich's Ataxia Neural Stem Cells.2019 · Front Cell Neurosci · In animals
  8. Mesenchymal stem cells improve motor functions and decrease neurodegeneration in ataxic mice.2015 · Mol Ther · In animals
  9. Generation and characterisation of Friedreich ataxia YG8R mouse fibroblast and neural stem cell models.2014 · PLoS One · In animals
  10. Unlocking Sulforaphane's Potential in Friedreich Ataxia: Further Evidence from Preclinical Investigations Using Induced Pluripotent Stem Cell-Derived Sensory Neurons.2026 · Antioxid Redox Signal · In the lab
  11. A phosphorylated variant of the mast/stem cell growth factor receptor KIT is upregulated in dorsal root ganglia of Friedreich ataxia.2026 · Histol Histopathol · In the lab
  12. Generation of Friedreich's ataxia induced pluripotent stem cells carrying the FXN c.165 + 5G>C splicing mutation.2026 · Stem Cell Res · In the lab
  13. Sulforaphane Targets Multiple Pathological Processes in Friedreich Ataxia Patient-Induced Pluripotent Stem Cell-Derived Sensory Neurons.2025 · Antioxid Redox Signal · In the lab
  14. Stem Cell Transplantation in Friedreich Ataxia: Cure for Leukemia but No Effect on Neurological Progression.2026 · Ann Clin Transl Neurol · Other paper
  15. Progress in understanding Friedreich's ataxia using human induced pluripotent stem cells.2019 · Expert Opin Orphan Drugs · Other paper
  16. Friedreich ataxia-induced pluripotent stem cell-derived neurons show a cellular phenotype that is corrected by a benzamide HDAC inhibitor.2016 · Hum Mol Genet · Other paper
  17. Mesenchymal stem cells restore frataxin expression and increase hydrogen peroxide scavenging enzymes in Friedreich ataxia fibroblasts.2011 · PLoS One · Other paper

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