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Updated: Jul 3, 2026

Modeling Charcot-Marie-Tooth Disease In Vitro by Transfecting Mouse Primary Motoneurons
Published on: January 7, 2019
Thrombopoietin is ineffective in a mouse model of motor neuron disease
Andrew Caraganis1, Susanna Benn, Merit Cudkowicz
1Day Neuromuscular Laboratory, Department of Neurology, Massachusetts General Hospital and Harvard Medical School, Charlestown, MA, USA.
Abstract:
This study assessed the therapeutic efficacy of thrombopoietin (TPO) in the mouse model of ALS using two treatment paradigms. TPO was administered either daily or in 13-day treatment cycles to SOD1-G93A mice. Quantitative analysis of platelet levels, VEGF and TGF-beta1 trophic factors were assessed. The effect of TPO on disease progression was analyzed by behavioral analysis and clinical examination. TPO treatment increased levels of platelets and TGF-beta1 but not VEGF. This treatment did not affect onset or survival in these mice. Although biologically active, demonstrated by increased platelet and TGF-beta1 levels, rmTPO did not attenuate disease progression in ALS mice.

