The treatment uses an approach called gene silencing.
So our bodies use a short strand of genetic code, called messenger RNA, to bridge the gap and carry the instructions.In acute intermittent porphyria it lowers the levels of an enzyme involved in haem production and prevents the build-up of toxic proteins.Prof David Rees, from King's College London, treated patients taking part in the trial in the UK.
Barry Greene, the president of Alnylam, which developed the porphyria drug, told the BBC the latest findings were"heralding a brand new class of medicine".went to the researchers who discovered RNA interference, which occurs naturally in our cells.Dr Alena Pance, from the Wellcome Sanger Institute, told the BBC News website:"I get excited about this, because targeting the messenger RNA allows the fine-tuning of the proteins that are involved in certain diseases.
This can be beneficial as it means you need treatment only once, but could also be more risky. If anything goes wrong, such as accidentally editing the wrong part of the genetic code, it cannot be undone.
George III will be pleased. However, have they discovered a cure for inbreeding?
'How much it will cost' is criminally poor reporting. How difficult a treatment is to develop or produce has NOTHING to do with its price. Soaking a person threatened with blindness or unendurable pain is abhorrent. Stop normalizing the profiteers of human misery.
you see we can do things right-get rid of all the greed is good brigade and we could achieve miracles,
Good news, but poor delivery. What sort of drug used is poorly explained. RNA? What sort of RNA? How does a it works? This new type of RNA (?) drug deserved much better reporting
How wonderful. I used to work in a unit specialising in Porphyria, and know how miserable and disruptive it can be.
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Source: The Mirror - 🏆 136. / 51 Read more »