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The Journal of Neuroscience, September 23, 2009, 29(38):11761-11771; doi:10.1523/JNEUROSCI.2734-09.2009

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Neurobiology of Disease
Motoneuron Transplantation Rescues the Phenotype of SMARD1 (Spinal Muscular Atrophy with Respiratory Distress Type 1)

Stefania Corti,^1,2 Monica Nizzardo,¹ Martina Nardini,¹ Chiara Donadoni,¹ Sabrina Salani,¹ Roberto Del Bo,¹ Dimitra Papadimitriou,¹ Federica Locatelli,³ Nicoletta Mezzina,¹ Francesca Gianni,¹ Nereo Bresolin,^1,2,3 and Giacomo P. Comi^1,2

¹Dino Ferrari Centre, Department of Neurological Sciences, University of Milan, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Foundation Ospedale Maggiore Policlinico, Mangiagalli and Regina Elena, 20122 Milan, Italy, ²Centre of Excellence on Neurodegenerative Diseases, University of Milan, 20122 Milan, Italy, and ³IRCCS Eugenio Medea, Bosisio Parini, 23842 Lecco, Italy

Correspondence should be addressed to Giacomo P. Comi, Department of Neurological Sciences, University of Milan, Istituto di Ricovero e Cura a Carattere Scientifico Foundation Ospedale Maggiore Policlinico, Mangiagalli and Regina Elena, Padiglione Ponti, Via Francesco Sforza 35, 20122 Milan, Italy. Email: giacomo.comi{at}unimi.it

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. We also 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.

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Received June 10, 2009; revised July 29, 2009; accepted Aug. 15, 2009.

Correspondence should be addressed to Giacomo P. Comi, Department of Neurological Sciences, University of Milan, Istituto di Ricovero e Cura a Carattere Scientifico Foundation Ospedale Maggiore Policlinico, Mangiagalli and Regina Elena, Padiglione Ponti, Via Francesco Sforza 35, 20122 Milan, Italy. Email: giacomo.comi{at}unimi.it

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