Gene silencing medicine transforms crippling pain


Senior Member
York, England
An innovative type of medicine - called gene silencing - is set to be used on the NHS for people who live in crippling pain.
The drug treats acute intermittent porphyria, which runs in families and can leave people unable to work or have a normal life.
Porphyria leads to a build-up of toxic proteins in the body, that cause the physical pain. Gene silencing "mutes" a set of genetic instructions to block that protein production.
"There's really nothing to stop us targeting so many different diseases from cancer to cardiovascular disease to cholesterol problems."

Gene silencing medicine transforms crippling pain - BBC News

Apparently acute intermittent porphyria is a rear disease and only 17 people a year are diagnosed with it in the U.K. If the medical community can do this, why can't they research and help the 240,000 U.K. ME/CFS sufferers!?


Senior Member
I think gene silencing and gene boosting is the future and I'm glad to read that novel technologies are also applied to rare diseases like this. Non-persistent gene therapies allow for much more precision medicine and preventative therapy to be applied in the future. It's also less risky than CRISPR and typical small molecule drugs that can always have different unpredicted cross-reactivities.