Transgenic-Mouse Model of Amyotrophic Lateral Sclerosis

Abstract

The article by Dr. Brown (Oct. 20 issue)¹ highlights the studies that my colleagues and I have performed with a transgenic model of human motor neuron disease.² Our experiments built on the studies of Dr. Brown and other members of a multicenter group that identified mutations of copper-zinc superoxide dismutase in some patients with a dominantly inherited form of amyotrophic lateral sclerosis.³ In mice expressing mutant forms of human copper-zinc superoxide dismutase a progressive paralytic disease develops that causes death at four to five months of age. Symptoms are accompanied by vacuolar changes, mitochondrial cytopathologic changes, alterations in the neuronal cytoskeleton, and the eventual degeneration of motor neurons, principally in the spinal cord.⁴