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Doctors treat porphyria using innovative gene silencing drug

Published on 15/04/19 at 10:56am

Doctors have successfully treated porphyria using an innovative ‘gene-silencing’ drug.

Speaking to the BBC, Professor David Rees, from King's College London, commented: “This is a really important treatment - it's innovative. Porphyria is one of the first conditions it has been used in successfully. I'm genuinely surprised how well it works in this condition and I think it offers a lot of hope for the future.”

The drug, called givosiran, kills messenger RNA - the short strands of genetic code used to communicate instructions.

Through a process called RNA interference givosiran prevents the production and build-up of toxic proteins responsible for the symptoms of porphyria.

Similar approaches could be used in treating other genetic conditions that cause nerve damage. Gene silencing has in the past been used to treat Huntington’s disease.

Dr Alena Pance, from the Wellcome Sanger Institute, said to the BBC: “I get excited about this, because targeting the messenger RNA allows the fine-tuning of the proteins that are involved in certain diseases.”

“And therefore, perhaps for the first time, [it can] offer a tool to be able to control those diseases to very accurate levels. There are diseases that are very difficult to find treatment for, that with this technology might be possible to tackle.”

Louis Goss

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