Spinal muscular atrophy is an infantile onset motor neuron disease caused by low SMN protein. The link between low SMN and selective motor neuron loss in the disease is still not clear. One means of e
A diploid 46(XX) human embryonic stem cell (HESC) line affected by Spinal muscular atrophy type 1 (SMA) due to homozygosity for a common deletion in the SMN1 gene was derived. By characterizing the me
