Stem Cell Transplantation for Spinal Cord Neurodegeneration

Amyotrophic lateral sclerosis (ALS), a disorder that affects 30,000 individuals in the USA alone, is characterized by relatively rapid degeneration of upper and lower motor neurons, with death normally occurring 2–5 years following diagnosis due to respiratory paralysis. Transplantation of various classes of neural precursor cells (NPCs) is a promising therapeutic strategy for the treatment of traumatic CNS injury and neurodegeneration, including ALS, because of the ability to replace lost or dysfunctional CNS cell types, provide neuroprotection, and deliver gene factors of interest. In order to target cellular therapy to diaphragmatic dysfunction in models of ALS, NPCs can be transplanted specifically into the cervical spinal cord ventral gray matter of both SOD1^G93A rats and mice. The SOD1^G93A rats and mice are currently the most well-studied animal model of the disease.