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A new strategy for the treatment of Wilson's disease

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Institute of Macromolecular Chemistry CAS

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The genetic disorder known as Wilson's disease is caused by a mutation of an ATP7B gene resulting in gradual hyperaccumulation of copper in the body. After reaching a certain concentration and depleting natural detoxication mechanisms the accumulated copper starts damaging the organism, especially the liver and brain. Wilson's disease cannot be cured, however, there are medications that aid the elimination of copper from the body. These medications must however be taken lifelong on a daily basis while the patients typically suffer from severe adverse side effects of such therapy. After years of research, the scientists at the Institute of Macromolecular Chemistry, Czech Academy of Sciences have come up with a treatment option minimizing these side effects by using copper scavenging directly within the digestive tract with a selectively copperchelating polymer. After oral administration, the polymer passes through the gastrointestinal tract where it picks up copper released from food and binds it firmly. The polymer with the bound copper is then eliminated from the body with stools.

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This video was supported by Strategy AV21 of the Czech Academy of Sciences (Research Program: Molecules and Materials for Life). The mission of the Czech Academy of Sciences (CAS) is top research focused on the problems and challenges of contemporary society.

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