US scientists have created the first human model for studying a devastating nerve disease, which allows them to watch how the disease develops and could help researchers find a way to treat it. Using skin cells from a child with spinal muscular atrophy, a genetic disease that attacks motor neurons in the spinal cord, researchers grew batches of nerve cells with the same genetic defects.
The findings allowed scientists to watch the nerve cells die off. The finding marks the latest advance in research that reprograms ordinary cell to look and act like embryonic stem cells. Infants with the most severe form the disease develop normally for the first six months of life, and then gradually develop muscle weakness and loss of motor control.
The team made motor neurons that contained the genetic defect that causes SMA with a powerful new type of cell called IPS cells, which behave like embryonic stem cells.
The findings allowed scientists to watch the nerve cells die off. The finding marks the latest advance in research that reprograms ordinary cell to look and act like embryonic stem cells. Infants with the most severe form the disease develop normally for the first six months of life, and then gradually develop muscle weakness and loss of motor control.
The team made motor neurons that contained the genetic defect that causes SMA with a powerful new type of cell called IPS cells, which behave like embryonic stem cells.