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from the mix-and-match-modular-theraputic-platforms dept.
Arthur T Knackerbracket has processed the following story:
Researchers from MIT, the McGovern Institute for Brain Research at MIT, the Howard Hughes Medical Institute, and the Broad Institute of MIT and Harvard have developed a new way to deliver molecular therapies to cells. The system, called SEND, can be programmed to encapsulate and deliver different RNA cargoes. SEND harnesses natural proteins in the body that form virus-like particles and bind RNA, and it may provoke less of an immune response than other delivery approaches.
The new delivery platform works efficiently in cell models, and, with further development, could open up a new class of delivery methods for a wide range of molecular medicines — including those for gene editing and gene replacement. Existing delivery vehicles for these therapeutics can be inefficient and randomly integrate into the genome of cells, and some can stimulate unwanted immune reactions. SEND has the promise to overcome these limitations, which could open up new opportunities to deploy molecular medicine.
[...] Reporting in Science, the team describes how SEND (Selective Endogenous eNcapsidation for cellular Delivery) takes advantage of molecules made by human cells. At the center of SEND is a protein called PEG10, which normally binds to its own mRNA and forms a spherical protective capsule around it. In their study, the team engineered PEG10 to selectively package and deliver other RNA. The scientists used SEND to deliver the CRISPR-Cas9 gene editing system to mouse and human cells to edit targeted genes.
[...] “By mixing and matching different components in the SEND system, we believe that it will provide a modular platform for developing therapeutics for different diseases,” said [CRISPR pioneer and senior study author Feng] Zhang.
Journal Reference:
Michael Segel, Blake Lash, Jingwei Song, et al. Mammalian retrovirus-like protein PEG10 packages its own mRNA and can be pseudotyped for mRNA delivery [$], Science (DOI: 10.1126/science.abg6155)
(Score: 2, Interesting) by ikanreed on Monday August 30 2021, @05:29PM (6 children)
But I'm not sure delivery mechanism side effects is the main ethical concern holding back CRISPR experiments in living people.
Then again recent evidence suggests I've clearly underestimated how much "I might have a slight rash at the injection site" dictates peoples' opinions about life saving treatments.
(Score: 3, Funny) by DannyB on Monday August 30 2021, @06:58PM (5 children)
1. Get an Emergency Use Authorization.
2. Get test subjects.
3. Begin therapeutic treatments
4. Begin looking for potential applications of this therapy.
5. Once a suitable medical condition is identified, begin research on how to integrate this delivery system with an active ingredient.
6. Begin new testing on new therapy for actual medical condition.
7. Repeat back to step 4 as many times as necessary, until . . .
8. Profit.
9. Get actual FDA approval.
10. More profit.
11. Produce data to show efficacy and safety, justifying the approval.
12. More profit.
If you eat an entire cake without cutting it, you technically only had one piece.
(Score: 2) by ikanreed on Monday August 30 2021, @08:18PM (4 children)
Yeah, but what's the emergency use for genetic alteration?
Most of the conditions I could see being treated with some early kind of gene therapy like Type I diabetes or hormone imbalances have really solid standard of care techniques that don't put patients at risk.
Even though the potential for improvement over SoC is really there, the risk dimensions are huge, because pretty much by definition the changes are permanent.
(Score: 2) by DannyB on Monday August 30 2021, @09:09PM
The emergency use is so that you can begin some sales.
Big pharmaceudakills.
If you eat an entire cake without cutting it, you technically only had one piece.
(Score: 2) by crafoo on Monday August 30 2021, @09:22PM (2 children)
I believe he was referring to COVID-19. Which is exactly what they did.
(Score: 2) by ikanreed on Tuesday August 31 2021, @01:30AM (1 child)
*sigh*
(Score: 2) by DannyB on Wednesday September 01 2021, @02:12PM
It was a joke. Formed around the idea that getting an emergency use authorization for covid was believed to be a bad idea by *cough* some people.
The joke has a different notion that big pharma values profit above all else, including killing their own customers.
If you eat an entire cake without cutting it, you technically only had one piece.
(Score: 3, Insightful) by Rosco P. Coltrane on Monday August 30 2021, @06:09PM (1 child)
(Score: 0, Disagree) by Anonymous Coward on Monday August 30 2021, @07:33PM
Or you could look at it this way, some of the most effective researchers also have a flair for public relations.
(Score: 2) by Snotnose on Monday August 30 2021, @08:53PM (1 child)
Just asking for a friend. Who is a huge fan of some old country song.
I came. I saw. I forgot why I came.
(Score: 2) by DannyB on Wednesday September 01 2021, @02:14PM
In The Wizard Of Oz (1939 movie), once they get into the emerald city, Dorothy asks: can you even dye my eyes to match my gown?
To which the helper nods in the affirmative.
If you eat an entire cake without cutting it, you technically only had one piece.
(Score: 2) by Beryllium Sphere (r) on Monday August 30 2021, @11:42PM
could it help with COVID vaccine production bottlenecks eventually?
The lipid coats used now are said to be extremely finicky work. Is it easier to manufacture injectable mRNA with this protein?
Of course it would have to go through clinical trials first, so quite a while before it could help.