Work

Microbial peptide de-coppers mitochondria: implications for Wilson disease

Year: 2016

Type: article

Abstract: The severe liver pathology of untreated Wilson disease (WD) is associated with massive copper overload caused by mutations in a liver-specific copper-transporting ATPase, ATP7B. While early, presymptomatic detection and chelation with conventional copper-binding molecules enables effective and life-saving treatment, liver transplantation is the sole option currently available for those with advanced disease. In this issue of the JCI, Lichtmannegger, Leitzinger, and colleagues delineate the therapeutic effect of methanobactin (MB), a potent bacterial copper-binding protein, at three late stages of disease in a WD rat model. Their results suggest that a formal clinical trial of MB in human subjects with severe hepatic pathology caused by WD would be rational.

Source: Journal of Clinical Investigation

DOI: 10.1172/jci88617

Author: Stephen G. Kaler

Language: English

Topic: Trace Elements in Health

Subfield: Nutrition and Dietetics

Field: Nursing

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: bronze

Funders: National Institutes of Health, National Heart, Lung, and Blood Institute, Eunice Kennedy Shriver National Institute of Child Health and Human Development

Awards: Disorders of Copper Transport, Choroid Plexus-Directed Gene Therapy for Alpha-Mannosidosis, Phase II Study of AAV9-GAA Gene Transfer in Pompe Disease, Effects of mutation of the LH/CG- receptor on its signaling activity, G9000128624