The APCs of neuroprotection.
Mutations in the enzyme superoxide dismutase 1 (SOD1) have been linked to the neurodegenerative disease amyotrophic lateral sclerosis (ALS). In this issue of the JCI, Zhong et al. report that the endogenous anticoagulant activated protein C (APC) is able to cross the blood-spinal cord barrier in mice and signal to both neuronal and non-neuronal cells (see the related article beginning on page 3437). This signaling resulted in the suppression of mutant SOD1 synthesis and retarded disease progression in a murine model of ALS. Here we discuss the potential importance of these data and possible relevance to human neurodegenerative diseases.
Cardiovascular Biology Research Program, Oklahoma Medical Research Foundation, and Howard Hughes Medical Institute, Oklahoma City, Oklahoma 73104, USA. Charles-Esmon@omrf.org
Charles T
CT
Esmon
Jonathan D
JD
Glass
D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy / Q000503:physiopathology; D000818:Animals; D000925:Anticoagulants / Q000378:metabolism / Q000494:pharmacology / Q000627:therapeutic use; D001812:Blood-Brain Barrier / Q000378:metabolism; D004195:Disease Models, Animal; D015971:Gene Expression Regulation, Enzymologic / Q000187:drug effects; D006801:Humans; D051379:Mice; D019636:Neurodegenerative Diseases / Q000188:drug therapy / Q000503:physiopathology*; D011486:Protein C / Q000378:metabolism* / Q000494:pharmacology / Q000627:therapeutic use; D015398:Signal Transduction
D000925:Anticoagulants; D011486:Protein C