Motoneuron Transplantation Rescues the Phenotype of SMARD1 (Spinal Muscular Atrophy with Respiratory Distress Type 1)
Articolo
Data di Pubblicazione:
2009
Citazione:
Motoneuron Transplantation Rescues the Phenotype of
SMARD1 (Spinal Muscular Atrophy with Respiratory
Distress Type 1) / S. Corti, M. Nizzardo, M. Nardini, C. Donadoni, S. Salani, R. Del Bo, D. Papadimitriou, F. Locatelli, N. Mezzina, F. Gianni, N. Bresolin, G.P. Comi. - In: THE JOURNAL OF NEUROSCIENCE. - ISSN 0270-6474. - 29:38(2009 Sep 23), pp. 11761-11771. [10.1523/JNEUROSCI.2734-09.2009]
Abstract:
Spinal muscular atrophy with respiratory distress type 1 (SMARD1) is a fatal form of infantile motoneuron disease. There is currently
no effective treatment, although motor neuron replacement is a possible therapeutic strategy. We transplanted purified motor neurons
into the spinal cord of nmd mice, an animal model of SMARD1.Wealso administered pharmacological treatment targeting the induction
of axonal growth toward skeletal muscle target. At the end stage of the disease, donor-derived motor neurons were detected in the nmd
anterior horns, extended axons into the ventral roots, and formed new neuromuscular junctions. These data correlated with improved
neuromuscular function and increased life spans. The neuroprotective effect was associated with a reduction in proinflammatory molecules
in treated spinal cords. This is the first report that functional restoration of motor units with transplanted motoneurons is feasible
in an animal model of a human motoneuron disease, opening up new possibilities for therapeutic intervention.
Tipologia IRIS:
01 - Articolo su periodico
Keywords:
SMARD1, Spinal muscular atrophy with respiratory distress type 1; motor neurons; stem cell; teraphy
Elenco autori:
S. Corti, M. Nizzardo, M. Nardini, C. Donadoni, S. Salani, R. Del Bo, D. Papadimitriou, F. Locatelli, N. Mezzina, F. Gianni, N. Bresolin, G.P. Comi
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