Amyotrophic Lateral Sclerosis

2011 Feb; 11(1) : 67-77.
Research advances in amyotrophic lateral sclerosis, 2009 to 2010.
Traub R, Mitsumoto H, Rowland LP.
Eleanor and Lou Gehrig MDA/ALS Research Center, Neurological Institute, Columbia University Medical
Center, 710 West 168th Street, New York, NY 10032, USA.
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease of upper and lower motor neurons that causes progressive weakness and death. The breadth of research in ALS continues to grow with exciting new discoveries in disease pathogenesis and potential future therapeutics. There is a growing list of identified mutations in familial ALS, including those in genes encoding TDP-43 and FUS/TLS, which are expanding our understanding of the role of RNA modulation in ALS pathogenesis. There is a greater
appreciation for the role of glial cells in motor neuron disease. Mitochondrial dysfunction is also being shown to be critical for motor neuron degeneration. In addition to pharmacotherapy, there are promising early developments with therapeutic implications in the areas of RNA interference, stem cell therapies, viral vector-mediated gene therapy, and immunotherapy. With greater understanding of ALS pathogenesis and exciting new therapeutic technologies, there is hope for future progress in treating this disease.
PMID: 21080240 [PubMed – in process]