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Crohn's Disease Forum » Books, Multimedia, Research & News » Knockout target against UC discovered


03-05-2016, 08:47 AM   #1
xeridea
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Knockout target against UC discovered

In a yet to be published study ("A protein truncating R179X variant in RNF186 confers protection against ulcerative colitis"), researchers have discovered a gene mutation that is protective against UC. If it passes review, this discovery will allow development of drugs that can knock out this gene and confer protection against UC.

This approach of targeting a condition like this is already being used in approved PCSK9 inhibitors that dramatically lower LDL cholesterol levels in patients. They found that people who naturally have really low LDL cholesterol levels have a mutation in the gene that produces the protein PCSK9. The PCSK9 protein binds to LDL receptor sites in the liver and breaks them down which prevents the liver from clearing LDL from body. The recently approved antibody drug Repatha knocks out the gene that produces PCSK9, clearing the way for the liver to slash LDL levels.

Similarly, if the research pans out, they can create an antibody against R179x that triggers a protective mechanism against UC. Of course this is still years out, but it's interesting that there are people looking at not just what genes may cause a condition, but also looking at the flip side of what genes may help protect against one.

This was a general IBD study looking at both UC and CD. Though initial findings favor UC, there may be something hidden in the data that can help CD in future.

Last edited by xeridea; 03-05-2016 at 12:32 PM.
03-06-2016, 04:26 AM   #2
fuzzy butterfly
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Hi thanks for that information. I don't have UC I have crohn's but hey if they can sort a cure for UC maybe crohn's might be next. Here's hoping for all IBD suffers.
Best wishes 💕
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