Posted on 09/13/2021 10:39:13 AM PDT by Red Badger
The United Kingdom's NHS has very recently approved a new cholesterol-lowering jab which will be offered to 300,000 people over the next three years.
The drug – inclisiran – will be administered twice a year as an injection.
It will mainly be prescribed to patients who suffer with a genetic condition that leads to high cholesterol, those who have already suffered a heart attack or stroke, or those who haven't responded well to other cholesterol-lowering drugs, such as statins.
There has been plenty of excitement surrounding the approval of the drug, both because of what it may be able to achieve, and because the drug uses a technique known as "gene silencing".
This is an emerging therapeutic technique that works by targeting the underlying causes of a disease, rather than the symptoms it causes. It does this by targeting a particular gene, and preventing it from making the protein that it produces.
Until now, most treatments using gene silencing technology have been used to treat rare genetic diseases. This means the cholesterol jab will be one of the first gene silencing drugs used to treat people on a wider scale.
Researchers are also currently investigating whether gene silencing could be used to treat a wide variety of health conditions, including Alzheimer's disease and cancer.
Gene silencing Gene silencing drugs work by targeting a specific type of RNA (ribonucleic acid) in the body, called "messenger" RNA. RNAs are found in every cell of the body, and play an important role in the flow of genetic information.
But messenger RNA (mRNA) is one of the most important types of RNA our body has, as it copies and carries genetic instructions from our DNA and makes specific proteins depending on the instructions.
In the case of the cholesterol jab, gene silencing works by targeting a protein called PCSK9 and degrading it. This protein is involved in regulating cholesterol in our bodies, but occurs in excess in people with high levels of LDL cholesterol (the "bad" cholesterol). Preventing this protein from being produced in the first place will reduce cholesterol levels.
In order to target this specific mRNA, researchers need to create a synthetic version of another type of RNA – called small interfering RNA (siRNA) – in the lab. This is a highly specific stretch of RNA which can be used to target specific mRNAs.
In this case, the siRNA is designed to specifically target the mRNA which carries instructions for the PCSK9 protein. It binds to its target mRNA and destroys the instructions, which significantly reduces the amount of these proteins that are produced.
Gene therapies are usually delivered using a viral vector – a virus-like vehicle that delivers genes to our cells in the same way a virus might infect them. So far, viral vector therapies have been used to treat rare genetic blood disorders, genetic blindness and spinal muscular atrophy.
Although viral vectors are very effective with one treatment, it may be impossible to deliver a second dose if needed due to adverse immune reactions. These drugs are also extremely costly.
Because of this, many of the gene silencing drugs currently being investigated are delivered using a different technique. Known as non-viral vector gene therapies, these deliver the drug using a nanoparticle which protects it from degradation in the blood so it can be delivered specifically to the target – such as the liver, which is the target of the cholesterol jab.
Gene silencing therapies delivered by non-viral vectors seem to hold more promise as they can be administered several times with limited side effects. Currently, non-viral vector therapies are used to treat a rare genetic condition called hereditary transthyretin-mediated amyloidosis, as well as in mRNA vaccines, such as BionTech/Pfizer and Moderna.
Interestingly, though, the cholesterol jab is not buried inside a nanoparticle or delivered with a viral vector.
Instead, the siRNA has been heavily modified in the lab to withstand degradation in the blood. It also has a ligand (a sugar molecule that works a bit like a hook) attached to it that allows it to specifically target liver cells.
Future treatments Several more gene silencing drugs are currently being investigated to treat a variety of other disorders, including in the kidney (such as preventing adverse reactions after a transplant), the skin (scarring), cancer (including melanoma, prostate, pancreatic, brain and other tumors) and eye disorders (such as age-related macular degeneration and glaucoma).
Researchers are also investigating whether gene silencing therapies could be useful in treating neurological and brain disorders, such as Huntington's disease and Alzheimer's disease.
Each of these gene silencing treatments would use similar techniques as other drugs that currently exist – by targeting a specific gene or protein and shutting it off. But in the case of cancer, because it's very complex, multiple different proteins may need to be targeted.
These gene silencing technologies will need to be shown to be effective in further clinical trials before they can be rolled out for use on a wider scale.
Another important challenge will be ensuring that the costs of these drugs remain low so many people can access them. But overall, these developments are very promising: gene silencing drugs are more specialized as they can target specific proteins in our cells.
This may be why they can be more successful in treating diseases than current treatments.
Aristides Tagalakis, Reader in Gene Delivery and Nanomedicine, Edge Hill University.
I officially trust no politicians, scientists, or doctors.
I’m going Neanderthal. Besides, I have an occipital bun...
Not surprised, but anyone who touts this as a good thing on FR should be keelhauled.
‘Gene Silencing’ is going to make the nuts nuttier.
I knew a guy had those all over his head.
His skull must have looked like a 3D map of West Virginia.................
LOL, I just have the one.
Before or after they are hung from the yardarm, drawn and quartered and made to walk the plank?..............
This is akin to sequestering co2 in terms of stupidity. The lipids hypothesis has been disproven. We need cholesterol, it repairs. And saying that cholesterol causes heart attacks is like saying that bleeding causes cuts.
what could possibly go wrong...
hehehe
I believe cholesterol is the precursor for all hormones, yes?
Just when we find out that Cholesterol is not the problem, they find a better way to get rid of it. High Cholesterol does not cause heart attacks. Plaque build up caused by metabolic syndrome does. Treat your liver well. Stay away from fructose. You don’t need medicine. You need a better diet.
My heart doctor tells me to eat more fruit!................
Now I know why!...................
CRISPR ❎
Hybridization. Nephilim
As in the Days of Noah
Matt 24:27
“I’m going Neanderthal.”
Correct. The healthiest people on the planet regarding long-term maladies (like diabetes, blood pressure, etc.) are those that basically eat like Neanderthals, including tribes in Africa and Eskimos in Alaska.
I have some old book by Weston A Price and studies on different “tribal” peoples.
“My heart doctor tells me to eat more fruit!................
Now I know why!”
Yes and no...not nearly as healthy as say, red meat, but still better than anything processed. The problem with fruits is the high sugar content, still better than eating carbs (for sure!) or HFCS, but green vegetables are the healthiest plants. Certain nuts are good too, if not overdone.
Same technology as our mRNA vaccines.
“I have some old book by Weston A Price and studies on different “tribal” peoples.”
I’ve read the story about the researcher who spent a year with a bunch of Eskimos in Alaska, and came back in PERFECT condition, even though virtually all he ate was meat.
Throw in some daily exercise for good measure and you've got it 100% right.
Yeah, one needs to eat the organ meat as well.
I think some tribes just eat the organs, and give the leaner meat to dogs.
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