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November 1, 2025Stem Cell ReportsOpen Access

Advances and challenges in modeling Charcot-Marie-Tooth type 2A using iPSC-derived models

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Authors

MRMafalda RizzutiEPElisa PagliariMDMartina D’Agostino

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Overview

Review highlights the potential and challenges of induced pluripotent stem cell models for understanding CMT2A and developing therapeutic strategies.

Key Points

  • This review assesses the current state of induced pluripotent stem cell models for Charcot-Marie-Tooth type 2A.
  • Review of existing iPSC-derived models of CMT2A
  • Analysis of studies focused on motor and sensory neuron differentiation
  • Discussion of pathogenetic insights from iPSC models
  • Identified challenges in utilizing iPSC for CMT2A research
  • Highlighted effective therapeutic strategies derived from iPSC models
  • Showed potential of iPSC models in understanding CMT2A disease mechanisms

Cite This Study

Rizzuti et al. (2025) studied this question.

synapsesocial.com/papers/69254371c0ce034ddc358996https://doi.org/10.1016/j.stemcr.2025.102711
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Also Consider

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

  1. 1Charcot–Marie‐Tooth type 2A in vivo models: Current updates2024 · 8 citations
  2. 2Generation and characterization of a patient-derived iPSC line, CSSi022-A (15666), with a pathogenic MFN2 mutation causing Charcot-Marie-Tooth disease type 2A2025
  3. 3Restoring the interplay between the endoplasmic reticulum and mitochondria by gene therapy improves Charcot–Marie–Tooth type 2A disease2026
  4. 4HDAC6 inhibition alleviates mitochondrial trafficking in novel models of Charcot-Marie-Tooth Disease Type 2A2026
  5. 5Pre-clinical Antisense Oligonucleotide Treatment of CMT2E in a Human Induced Pluripotent Stem Cell (iPSC)-derived Motor Neuron Model (S5.008)2024