Affecting at least 14,000 Americans, amyotrophic lateral sclerosis (ALS) is a paralyzing and highly fatal neurodegenerative disorder for which there are no effective treatments. Scientists peered inside neurons and watched the workings of annexin A11, a gene linked to a rare form of ALS. They found that neurons may normally use the gene to ship internal housekeeping instructions via a newly discovered “hitchhiking” system and that disease-causing mutations may tie up deliveries at the cell’s loading docks. The study, published in Cell, was led by researchers in labs overseen by Michael Ward, M.D., Ph.D., investigator at the National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health; Jennifer Lippincott-Schwartz, Ph.D., senior group leader at the Howard Hughes Medical Institute Janelia Research Campus; and Peter St. George-Hyslop, M.D., Wellcome Trust Principal Research Fellow, Cambridge Institute for Medical Research., United Kingdom.
