NeuroscienceInternational Journal of Molecular SciencesPeer-reviewed
Muscle-specific DNM2 overexpression improves Charcot-Marie-Tooth disease in vivo and reveals a narrow therapeutic window in skeletal muscle
Marie Goret, Gwenaelle Piccolo, Jocelyn Laporte
This study shows that muscle-specific DNM2 overexpression improves CMT symptoms in mice, but systemic delivery worsens muscle pathology, highlighting a narrow therapeutic window for DNM2 modulation.
DOI 10.3390/ijms27031471