Amyotrophic lateral sclerosis: update and new developments

Ashley J Pratt¹, Elizabeth D Getzoff¹, J Jefferson P Perry^1,2
1Department of Molecular Biology and The Skaggs Institute for Chemical Biology, The Scripps Research Institute, La Jolla, CA 92037, USA; ²The School of Biotechnology, Amrita University, Kollam, Kerala 690525, India

Abstract: Amyotrophic lateral sclerosis (ALS) is the most common form of motor neuron disease. It is typically characterized by adult-onset degeneration of the upper and lower motor neurons, and is usually fatal within a few years of onset. A subset of ALS patients has an inherited form of the disease, and a few of the known mutant genes identified in familial cases have also been found in sporadic forms of ALS. Precisely how the diverse ALS-linked gene products dictate the course of the disease, resulting in compromised voluntary muscular ability, is not entirely known. This review addresses the major advances that are being made in our understanding of the molecular mechanisms giving rise to the disease, which may eventually translate into new treatment options.

Keywords: amyotrophic lateral sclerosis, neurodegeneration, motor neuron disease, genetics, aging