Using genetically modified stem cells to halt the progression of ALS

(ALS) is a neurodegenerative disease characterized by the loss of motor neurons leading to paralysis and death. The vast majority of ALS cases are idiopathic; however, at least 2% are caused by mutation of the copper-zinc superoxide dismutase 1 gene on chromosome 21. Here, we propose a three-pronged approach: (1) identify the molecular trigger for the onset of symptomatic ALS using a microray approach, (2) develop a genetically modified cell-based treatment, and (3) restore lost respiratory function once disease progression has been halted by an implanted stem cell treatment.