Neural stem cell-derived motoneurons for treatment of spinal muscular atrophy

The aim of the study is to explore the therapeutic potential of transplantation of differentiated motor neurons into the spinal cord of mice presenting neuromuscular degeneration (nmd). Purified stem cell-derived motor neurons were transplanted into the spinal cord of nmd mice, an animal model of spinal muscular atrophy with respiratory distress type 1 (SMARD1). The transplantation ameliorated the nmd phenotype.