[PDF][PDF] ALS: a disease of motor neurons and their nonneuronal neighbors

S Boillée, CV Velde, DW Cleveland - Neuron, 2006 - cell.com
S Boillée, CV Velde, DW Cleveland
Neuron, 2006cell.com
Amyotrophic lateral sclerosis is a late-onset progressive neurodegenerative disease
affecting motor neurons. The etiology of most ALS cases remains unknown, but 2% of
instances are due to mutations in Cu/Zn superoxide dismutase (SOD1). Since sporadic and
familial ALS affects the same neurons with similar pathology, it is hoped that therapies
effective in mutant SOD1 models will translate to sporadic ALS. Mutant SOD1 induces non-
cell-autonomous motor neuron killing by an unknown gain of toxicity. Selective vulnerability …
Amyotrophic lateral sclerosis is a late-onset progressive neurodegenerative disease affecting motor neurons. The etiology of most ALS cases remains unknown, but 2% of instances are due to mutations in Cu/Zn superoxide dismutase (SOD1). Since sporadic and familial ALS affects the same neurons with similar pathology, it is hoped that therapies effective in mutant SOD1 models will translate to sporadic ALS. Mutant SOD1 induces non-cell-autonomous motor neuron killing by an unknown gain of toxicity. Selective vulnerability of motor neurons likely arises from a combination of several mechanisms, including protein misfolding, mitochondrial dysfunction, oxidative damage, defective axonal transport, excitotoxicity, insufficient growth factor signaling, and inflammation. Damage within motor neurons is enhanced by damage incurred by nonneuronal neighboring cells, via an inflammatory response that accelerates disease progression. These findings validate therapeutic approaches aimed at nonneuronal cells.
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