RT Journal Article
T1 Small GSK-3 Inhibitor Shows Efficacy in a Motor Neuron Disease Murine Model Modulating Autophagy
A1 Munk, Estefanía de
A1 Palomo, Valle
A1 Muñoz Sáez, Emma
A1 Pérez, Daniel I.
A1 Gómez Miguel, Begoña
A1 Solas Alados, Mª Teresa
A1 Gil, Carmen
A1 Martínez, Ana
A1 Arahuetes Portero, Rosa María
AB Amyotrophic lateral sclerosis (ALS) is a progressive motor neuron degenerative disease that has no effective treatment up to date. Drug discovery tasks have been hampered due to the lack of knowledge in its molecular etiology together with the limited animal models for research. Recently, a motor neuron disease animal model has been developed using β-Nmethylamino-L-alanine (L-BMAA), a neurotoxic amino acid related to the appearing of ALS. In the present work, the neuroprotective role of VP2.51, a small heterocyclic GSK-3 inhibitor, is analysed in this novel murine model together with the analysis of autophagy. VP2.51 daily administration for two weeks, starting the first day after L-BMAA treatment, leads to total recovery of neurological symptoms and prevents the activation of autophagic processes in rats. These results show that the L-BMAA murine model can be used to test the efficacy of new drugs. In addition, the results confirm the therapeutic potential of GSK-3 inhibitors, and specially VP2.51, for the disease-modifying future treatment of motor neuron disorders like ALS.
PB Public Library of Sciences (PLOS)
SN 1932-6203
YR 2016
FD 2016-09-15
LK https://hdl.handle.net/20.500.14352/17606
UL https://hdl.handle.net/20.500.14352/17606
LA eng
NO Ministerio de Economía y Competitividad (MINECO)
NO Association Francaise contre les Myopathies
NO FUNDELA (Fundación Española para el Fomento de la Investigación de la Esclerosis Lateral Amiotrófica.)
DS Docta Complutense
RD 21 abr 2025