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October 18, 2025Open Access

Gene therapy-mediated overexpression of wild-type MFN2 improves Charcot-Marie-Tooth disease type 2A

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Authors

MTM. TessierAix-Marseille UniversitéZHZeinab HamzéAix-Marseille UniversitéNBNathalie Bonello‐PalotAix-Marseille Université

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Overview

Gene therapy shows improved mitochondrial dynamics and motor function in CMT2A mice, suggesting new treatment possibilities.

Key Points

  • Gene therapy using MFN2 leads to significant improvements in motor function in CMT2A mice.
  • AAV9 vector delivering wild-type MFN2 effectively restores mitochondrial function and neuronal integrity.
  • Therapeutic benefits were observed even after the onset of symptoms, reversing molecular markers of CMT2A.
  • The treatment was well tolerated, without major adverse effects like hepatotoxicity or inflammation.

Cite This Study

Tessier et al. (2025) studied this question.

synapsesocial.com/papers/68f408995de60f8893c6ff2fhttps://doi.org/10.1101/2025.10.15.682364
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Also Consider

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

  1. 1Restoring the interplay between the endoplasmic reticulum and mitochondria by gene therapy improves Charcot–Marie–Tooth type 2A disease2026
  2. 2Neuromuscular Dysfunction and Charcot-Marie-Tooth Disease Reversal in Mfn2 T105M Knock-In Rats2026
  3. 3AAVrh74.tMCK.NT-3 Surrogate Gene Therapy in a Mouse Model of CMT2A2026
  4. 4A cellular assay to determine the fusion capacity of MFN2 variants linked to Charcot-Marie-Tooth type 2A2024
  5. 5HDAC6 inhibition alleviates mitochondrial trafficking in novel models of Charcot-Marie-Tooth Disease Type 2A2026