Skip to main content

Thank you for visiting You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

Schwann cell gene therapies in sight

This is a preview of subscription content

Access options

Rent or Buy article

Get time limited or full article access on ReadCube.


All prices are NET prices.


  1. 1.

    Kagiava A, Karaiskos C, Richter J, Tryfonos C, Jennings MJ, Heslegrave AJ, et al. AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy. Gene Ther. 2021. In press.

  2. 2.

    Skre H. Genetic and clinical aspects of Charcot–Marie–Tooth’s disease. Clin Genet. 1974;6:98–118.

    CAS  Article  Google Scholar 

  3. 3.

    Fridman V, Bundy B, Reilly MM, Pareyson D, Bacon C, Burns J, et al. CMT subtypes and disease burden in patients enrolled in the Inherited Neuropathies Consortium natural history study: a cross-sectional analysis. J Neurol Neurosurg Psychiatry. 2015;86:873–8.

    CAS  Article  Google Scholar 

  4. 4.

    Scherer SS, Xu YT, Messing A, Willecke K, Fischbeck KH, Jeng LJ. Transgenic expression of human connexin32 in myelinating Schwann cells prevents demyelination in connexin32-null mice. J Neurosci. 2005;25:1550–9.

    CAS  Article  Google Scholar 

  5. 5.

    Abel A, Bone LJ, Messing A, Scherer SS, Fischbeck KH. Studies in transgenic mice indicate a loss of connexin32 function in X-linked Charcot–Marie–Tooth disease. J Neuropathol Exp Neurol. 1999;58:702–10.

    CAS  Article  Google Scholar 

  6. 6.

    Jeng LJ, Balice-Gordon RJ, Messing A, Fischbeck KH, Scherer SS. The effects of a dominant connexin32 mutant in myelinating Schwann cells. Mol Cell Neurosci. 2006;32:283–98.

    CAS  Article  Google Scholar 

  7. 7.

    Sargiannidou I, Vavlitou N, Aristodemou S, Hadjisavvas A, Kyriacou K, Scherer SS, et al. Connexin32 mutations cause loss of function in Schwann cells and oligodendrocytes leading to PNS and CNS myelination defects. J Neurosci. 2009;29:4736–49.

    CAS  Article  Google Scholar 

  8. 8.

    Scherer SS, Xu YT, Nelles E, Fischbeck K, Willecke K, Bone LJ. Connexin32-null mice develop demyelinating peripheral neuropathy. Glia. 1998;24:8–20.

    CAS  Article  Google Scholar 

  9. 9.

    Bailey RM, Rozenberg A, Gray SJ. Comparison of high-dose intracisterna magna and lumbar puncture intrathecal delivery of AAV9 in mice to treat neuropathies. Brain Res. 2020;1739:146832.

    CAS  Article  Google Scholar 

  10. 10.

    Bailey RM, Armao D, Nagabhushan Kalburgi S, Gray SJ. Development of intrathecal AAV9 gene therapy for giant axonal neuropathy. Mol Ther Methods Clin Dev. 2018;9:160–171.

    CAS  Article  Google Scholar 

Download references

Author information



Corresponding author

Correspondence to Stephan Züchner.

Ethics declarations

Conflict of interest

The author declares no competing interests.

Additional information

Publisher’s note Springer Nature remains neutral with regard to jurisdictional claims in published maps and institutional affiliations.

Rights and permissions

Reprints and Permissions

About this article

Verify currency and authenticity via CrossMark

Cite this article

Züchner, S. Schwann cell gene therapies in sight. Gene Ther 28, 618–619 (2021).

Download citation


Quick links