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@ARTICLE{Yuan:278040,
      author       = {Yuan, Xidi and Klein, Dennis and Maier, Anna-Maria and
                      Martini, Rudolf},
      title        = {{T}herapeutic sphingosine-1-phosphate receptor modulation
                      by repurposing fingolimod ({FTY}720) leads to mitigated
                      neuropathy and improved clinical outcome in a mouse model
                      for {C}harcot-{M}arie-{T}ooth 1{X} disease.},
      journal      = {Neuromuscular disorders},
      volume       = {50},
      issn         = {0960-8966},
      address      = {Amsterdam [u.a.]},
      publisher    = {Elsevier Science},
      reportid     = {DZNE-2025-00546},
      pages        = {105345},
      year         = {2025},
      abstract     = {Previous studies have shown that both the innate and
                      adaptive immune systems foster progression of neuropathy and
                      clinical symptoms in a mouse model for Charcot-Marie-Tooth
                      1X disease. Here we demonstrate a possible therapeutic
                      translation of these findings using the clinically approved
                      sphingosine-1-phosphate receptor modulator fingolimod
                      (FTY720) in connexin32-deficient mice mimicking
                      Charcot-Marie-Tooth 1X disease. Treatment with FTY720
                      prevented an increase of CD8+ and CD4+ T-lymphocyte numbers
                      in both femoral quadriceps nerve as well as in ventral
                      spinal roots. While macrophages of ventral spinal roots show
                      a similar, albeit non-significant trend, macrophages from
                      quadriceps nerve are not reduced upon treatment. On the
                      histopathological level, axonopathic changes were reduced in
                      ventral spinal roots, but not in quadriceps nerves upon
                      treatment. Electrophysiological recordings displayed
                      improved nerve conduction parameters upon FTY720 treatment,
                      while clinically, FTY720 treatment ameliorated distinct
                      parameters of motor performance and grip strength. We
                      suggest that targeting the adaptive immune system might be a
                      pharmacological treatment option for mitigating disease
                      burden particularly in severe cases of Charcot-Marie-Tooth
                      1X.},
      keywords     = {Axon damage (Other) / FTY720 (Other) / Fingolimod (Other) /
                      Inherited peripheral neuropathy (Other) / Macrophages
                      (Other) / Myelin (Other) / Neuroinflammation (Other) /
                      T-lymphocytes (Other)},
      cin          = {AG Neher (München)},
      ddc          = {610},
      cid          = {I:(DE-2719)1110011},
      pnm          = {352 - Disease Mechanisms (POF4-352)},
      pid          = {G:(DE-HGF)POF4-352},
      typ          = {PUB:(DE-HGF)16},
      pubmed       = {pmid:40239532},
      doi          = {10.1016/j.nmd.2025.105345},
      url          = {https://pub.dzne.de/record/278040},
}