Since the anti-science foo-foo hippies lost their bid to have all foods labeled ‘CONTAINS GMO WARBLEGARBLE! TEACH THE CONTROVERSY!’, a group of scientists at the Evil League of Evil have generated GMO corn for LSD. I guess as a gesture of good-will, or something.
I hope everyones happy now.
The GMO corn for ‘LSD’ is not for the mind-altering drug ‘LSD’, but actually a putative treatment option for ‘Lysosomal storage diseases‘? This is GMO corn that could help treat a collection of genetic conditions that kill children/debilitate adults?
Oh thats much cooler!!
People with a lysosomal storage disease have… something ‘wrong’ with their lysosomes. When your cells cannot break down waste/old proteins/etc properly, all that garbage builds up, and terrible things happen. You might have heard of Tay–Sachs disease (‘common’ in European Jewish populations), or Niemann–Pick disease. Usually these kinds of diseases arise from non-functional or disfunctional enzymes (proteins that *do* something, have an active ‘job’).
Thus some of these diseases can be treated with enzyme replacement therapy… to the tune of $300,000-$800,000 per year.
Why the hell is this treatment so expensive? I mean people with Type I diabetes also need a constant infusion of a protein, insulin, and they only cost maybe a couple thousand bucks to treat a year. Why $800,000???
Because enzymes are hard to produce cheaply.
One way we cut costs is to grow needed proteins in bacteria, blow up the bacteria, harvest the protein. We cant do that with enzymes humans need, though, because in order for the enzyme to work properly and not illicit an immune response, it needs to be decorated properly. With sugar. And bacteria dont use the same sugars human use. Our immune system uses this fact to fight bacteria.
We also cant get plants/plant cell lines to make the enzymes for us– They have sugars humans dont have too– xylose and fucose.
So scientists have been working really hard trying to figure out a way to grow the necessary enzymes in bacteria/plants that keep the ‘bad’ sugars from being added– but they all require modifying the enzyme in some way. Too many modifications, and you risk generating an immune response to the enzyme when you try to administer it to patients.
This group of scientists were very, very clever.
A lot of the sugar ‘decorations’ are changed in a cells Golgi. So, an enzyme being made in a plant cell in the endoplasmic reticulum, and the same enzyme being made in a mammalian cell in the endoplasmic reticulum, look pretty much the same. But when those same proteins are trafficked to the Golgi, the plant enzyme gets xylose and fucose decorations, and the mammalian enzyme gets galactose and sialic acid decorations.
They noticed that a protein in plants, γ-Zein, is made in the ER, and then goes about its business. It doesnt get shuffled over to the Golgi. And this is the result of the mRNA that makes γ-Zein, NOT a feature of the γ-Zein protein itself.
Which means that they could, theoretically, put the γ-Zein “GO TO THE ER AND THEN DO NOOOOT GO TO THE GOLGI!!!” message on an enzyme we want to make for and LSD, and maybe we could get the plants to make a human-useable enzyme, without altering the protein in any way!
They tried it with one of the enzymes used to treat MPS I, α-L-iduronidase. They generated a GMO corn that would make human α-L-iduronidase with the corn γ-Zein mRNA signals to make sure it stayed in the ER and didnt get ‘decorated’ in the Golgi.
And it worked!
The corn made ‘good’ human α-L-iduronidase! The right sugar on the outside, it appeared to be biologically active (even though they didnt test it in any animal models or anything, just in lab experiments). There was some contamination by α-L-iduronidase with ‘bad’ sugars– the mRNA signal was not able to keep ALL of the protein out of the Golgi– but it was easy enough to clean up the protein by filtering out any of it that ‘stuck’ to anti-xylose or fucose purification schemes.
And again, none of this has made it into humans yet. And, just to be clear, its not like LSD patients will be able to eat corn instead of getting IV infusions of functioning enzymes.
What this study is, is showing that there might be a cheaper, safer (dont have to worry about human pathogens contaminating a corn field!) way of treating a family of genetic diseases.