Article
IPLEX administration improves motor neuron survival and ameliorates motor functions in a severe mouse model of spinal muscular atrophy.
Molecular medicine (Cambridge, Mass.) - 25 Sept 2012
Murdocca Michela, Malgieri Arianna, Luchetti Andrea, Saieva Luciano, Dobrowolny Gabriella, de Leonibus Elvira, Filareto Antonio, Quitadamo Maria Chiara, Novelli Giuseppe, Musarò Antonio, Sangiuolo Federica
Abstract excerpt
Spinal muscular atrophy (SMA) is an inherited neurodegenerative disorder and the first genetic cause of death in childhood. SMA is caused by low levels of survival motor neuron (SMN) protein that induce selective loss of α-motor neurons (MNs) in the spinal cord, resulting in progressive muscle at...
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