Research on new and existing medicines for Wilson’s disease

Trientine

Trientine is one of the most common treatments for Wilson’s disease. Current research is looking at ways to improve how it is used. 

Methanobactin

A possible new medicine is methanobactin. This comes from bacteria and can bind copper. In animal tests it was able to remove copper build-up in the liver in around 8 days. Researchers hope  this could lead to treatments that do not need to be taken all the time.

But lots more testing is needed to confirm it works and is safe in humans.

Genetic treatments and gene therapy for Wilson’s disease

Genetic treatments for many diseases are developing very quickly. Wilson’s disease could be very well suited to these new treatments.

We know that Wilson’s disease is caused by a problem with just one gene not working properly. So it could be possible to target this gene in liver cells to treat or even cure Wilson’s disease.

There are several genetic therapy approaches being explored but these are in the very early stages and have not yet had much testing.

Adding a normal version of the gene

This treatment uses a safe virus to carry a healthy copy of the Wilson’s disease gene into the liver. The new gene helps the body make the Wilson’s protein.

Early tests have taken place and more are planned. But it may be years before we know whether this gene therapy can help more people.

Gene editing for Wilson’s disease

Some newer genetic treatments use “genetic scissors” called CRISPR. These gene editing tools can go into a cell and snip out, replace, or repair a faulty gene.

Some very early tests have been done in other conditions using this type of treatment. One day it might be possible to use this to repair the Wilson’s disease gene in the liver. But a lot more research is needed to see if it works and is safe.

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Take part in research

Researchers from the University of Birmingham, are seeking individuals with Wilson disease to participate in an online survey.

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