PT  - JOURNAL ARTICLE
AU  - Stefania Corti
AU  - Monica Nizzardo
AU  - Martina Nardini
AU  - Chiara Donadoni
AU  - Sabrina Salani
AU  - Roberto Del Bo
AU  - Dimitra Papadimitriou
AU  - Federica Locatelli
AU  - Nicoletta Mezzina
AU  - Francesca Gianni
AU  - Nereo Bresolin
AU  - Giacomo P. Comi
TI  - Motoneuron Transplantation Rescues the Phenotype of SMARD1 (Spinal Muscular Atrophy with Respiratory Distress Type 1)
AID  - 10.1523/JNEUROSCI.2734-09.2009
DP  - 2009 Sep 23
TA  - The Journal of Neuroscience
PG  - 11761--11771
VI  - 29
IP  - 38
4099  - http://www.jneurosci.org/content/29/38/11761.short
4100  - http://www.jneurosci.org/content/29/38/11761.full
SO  - J. Neurosci.2009 Sep 23; 29
AB  - 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.