Stem cell treatment & Charcot-Marie-Tooth disease

Written based on 17 research studies on PubMed, searched on 14 Nov 2026.

The claim

Posts online say that stem cell treatment helps with Charcot-Marie-Tooth disease.

Where research stands

We found 32 published papers on stem cell treatment and Charcot-Marie-Tooth disease. They were published between 2008 and 2026. 1 of them is a study in people. None of them compared people who got it with a group that did not. 8 were studies in animals, and 16 were only in cells in the lab. 2 papers are reviews that sum up other studies. 1 new study in people is running or will start soon, say the trial registers. 6 research projects on stem cell treatment for Charcot-Marie-Tooth disease are funded and still running.

• 1In people
• 8In animals
• 16Only 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

Sources

1. A Phase 1 clinical trial to evaluate the safety and tolerability of CLZ-2002 for the treatment of patients with Charcot-Marie-Tooth disease type 1.2026 · Stem Cells Transl Med · Study in people
2. Establishment of an In Vitro Disease Model of Charcot-Marie-Tooth Disease using Human Induced Pluripotent Stem Cells.2025 · Juntendo Med J · Review
3. Immune effects of mesenchymal stem cells: implications for Charcot-Marie-Tooth disease.2008 · Cell Immunol · Review
4. Synergistic effect of Wharton's jelly-derived mesenchymal stem cells and insulin on Schwann cell proliferation in Charcot-Marie-Tooth disease type 1A treatment.2024 · Neurobiol Dis · In animals
5. Preclinical Efficacy of Peripheral Nerve Regeneration by Schwann Cell-like Cells Differentiated from Human Tonsil-Derived Mesenchymal Stem Cells in C22 Mice.2023 · Biomedicines · In animals
6. Cytokines secreted by mesenchymal stem cells reduce demyelination in an animal model of Charcot-Marie-Tooth disease.2022 · Biochem Biophys Res Commun · In animals
7. Stem Cell Therapy Enhances Motor Activity of Triceps Surae Muscle in Mice with Hereditary Peripheral Neuropathy.2021 · Int J Mol Sci · In animals
8. Fibulin 5, a human Wharton's jelly-derived mesenchymal stem cells-secreted paracrine factor, attenuates peripheral nervous system myelination defects through the Integrin-RAC1 signaling axis.2020 · Stem Cells · In animals
9. Differentiation of Human Tonsil-Derived Mesenchymal Stem Cells into Schwann-Like Cells Improves Neuromuscular Function in a Mouse Model of Charcot-Marie-Tooth Disease Type 1A.2018 · Int J Mol Sci · In animals
10. [Generation of induced pluripotent stem cells from peripheral blood mononuclear cells of a patient with autosomal recessive Charcot-Marie-Tooth disease type 2S caused by IGHMBP2 mutations].2025 · Zhong Nan Da Xue Xue Bao Yi Xue Ban · In the lab
11. Advances in modeling the Charcot-Marie-Tooth disease: Human induced pluripotent stem cell-derived Schwann cells harboring SH3TC2 variants.2025 · Eur J Cell Biol · In the lab
12. Generation of two induced pluripotent stem cell lines from Charcot-Marie-Tooth type 1B patients harboring autosomal dominant mutations in myelin protein zero gene.2025 · Stem Cell Res · In the lab
13. Establishment of a human induced pluripotent stem cell (iPSC) line (JUCTCi018-A) from a patient with Charcot-Marie-Tooth disease type 2EE (CMT2EE) due to a homozygous c.122G > A p.(Arg41Gln) mutation in the MPV17 gene.2024 · Stem Cell Res · In the lab
14. The Regentime stem cell procedure, successful treatment for a Charcot-Marie-Tooth disease case.2024 · Clin Case Rep · Other paper
15. Corrigendum to "Generation of induced pluripotent stem cell line, ZJUCHi002-A, from Charcot-Marie-Tooth disease type 2A (CMT2A) patient with a mutation of c.752C>T in MFN2" [Stem Cell Res. 36C (2019) 101411].2023 · Stem Cell Res · Other paper
16. Corrigendum to "Generation of a human Charcot-Marie-Tooth disease type 1B (CMT1B) iPSC line, ZJUCHi001-A, with a mutation of c.292C>T in MPZ" [Stem Cell Res. 35C (2019) 101407].2023 · Stem Cell Res · Other paper
17. HDAC6 Inhibition Corrects Electrophysiological and Axonal Transport Deficits in a Human Stem Cell-Based Model of Charcot-Marie-Tooth Disease (Type 2D).2022 · Adv Biol (Weinh) · Other paper