TABLE 1.

List of Neurodegenerative Diseases That Can Be Impacted by Cell-and/or Gene-Based Therapies

Disease	Underlying Cause	Potential Treatment Strategy
PD	Death of dopaminergic neurons	Transplantation of dopaminergic neurons derived from ESC/iPS cells
Stroke	Neuronal death	Cell replacement with fully functional neurons derived from ESC/iPS cells
ALS	Loss of motor neurons	Cell therapy based on motor neurons derived from ESC/iPS cells
SMA	Spinal motor neuron death	Drug screening based on in vitro models of NPs/iPS cells for SMA; generation of spinal motor neurons from iPS cells
HD	Death of neurons in the striatum	Cell replacement with striatal neurons derived from iPS cells
AD	Loss of neurons and synapses throughout the brain	Drug screening based on in vitro models of AD; gene and cell therapy: transplantation of cells that overexpress NGF
MS	Immune system attacking myelin sheaths	Cell therapy based on transplantation of oligodendrocytes derived from ESC/iPS cells; autologous stem cell transplantation to repopulate the immune system

iPS, induced pluripotent stem