Neuroprotection in Spinal Muscular Atrophy (SMA) using neural stem cells as a therapeutic approach

The project objective is to contribute to the development of a stem cell therapy for Spinal Muscular

Atrophy (SMA). SMA is a common motor neuron degeneration disorder, manifesting with progressive

muscle weakness and paralysis and leading to death. It is caused by mutations in the survival motor

neuron gene.

We will investigate the therapeutic potential of transplanted neural stem cells generated from human

adult reprogrammed somatic stem cells (induced pluripotent stem cells) to ameliorate the phenotype of

an SMA mouse model. We will also use stem cells that are genetically modified to secrete growth

factors and become more supportive to the motor neurons that die in SMA. The cells will be

transplanted into the spinal cord of SMA mice, and we will evaluate whether and how their

engraftment modifies disease progression.

These experiments in the SMA mouse model explore the feasibility of stem cell therapy for SMA and

other motor neuron diseases.