Theoretically, all kinds of gene mutations can be corrected by gene editing, but there may be a long way to go before this ultimate killer can fully play its role. Therefore, it may be more realistic to solve, or even partially solve, the problem of gene mutation by using existing technologies and means.
The problem is that just as people only care about online celebrities and small meat, researchers are mainly interested in those popular genes. This is not entirely because they just want to pursue fame and fortune, but because of reality. To engage in unpopular genes is to sit on the bench yourself. Perhaps plum blossom fragrance comes from bitter cold, but it is difficult for researchers not to freeze to death and live to the day of success.
The same is true of genetic research on autism. If there are many genes that affect patients, researchers will be bustling and there will be more research data. Some people will try their best to experiment with the existing means, and may be the first to appear a treatment. The patient's few genes will inevitably come to her door, and there are fewer and fewer cars and horses, and no one cares about it for several years, let alone treatment. No one even studies why this gene causes autism.
Among many autism genes, PTEN gene is a wonderful flower.
There are many autistic patients with PTEN gene mutation, which may account for 2%~5%. What is more striking is that PTEN gene is a very important tumor suppressor gene. In fact, PTEN is the second best tumor suppressor gene, except that it is so hot that people just want to vomit. Mutations in PTEN gene were found in as many as 30% of tumor cells. We know that cancer research is twice as popular as autism research-the bosses in charge of funds are afraid of cancer, and they are obviously beyond the age of autism. Therefore, the study of PTEN and autism has also touched a little light.
How PTEN gene mutation causes autism is not detailed here. As long as we know, as a tumor suppressor gene, the product of PTEN gene, PTEN protein, its function is to prevent cells from growing and differentiating endlessly-equivalent to a brake, that's all.
People born with PTEN gene mutation are equivalent to having a brake failure in their bodies. Of course, because there are other brakes, the situation is not completely out of control; However, under unfortunate circumstances, some body cells may get out of control and grow in danger, including brain nerve cells. For example, the terrible glioblastoma is caused by the mutation of PTEN gene. Autistic patients with PTEN gene mutation usually have symptoms of megacephaly.
Generally speaking, patients with PTEN gene mutation, whether autistic or tumor patients, the root of the problem is that the number of PTEN protein in the body is not enough or can not work normally. The solution to the problem is simple, that is, to find a way to keep this brake.
How to save it?
Gene editor, the ultimate killer, not to mention it. At present, one of the methods being studied is to send good PTEN gene into body cells with some vector to increase the number of PTEN protein.
In other words, many patients still have a good PTEN gene, so because there is a phenomenon of "cutting off the beard" in the process of gene transfer to protein, we can try our best to prevent "cutting off the beard", thus increasing the output of PTEN protein. At present, this operation is completely feasible in theory, and there are some studies that use miRNA popular in previous years.
Or, roughly speaking, make PTEN protein directly in the test tube, and then try to get it into the cell. However, this method may be the most difficult because it is difficult for protein to cross the cell membrane.
Or ... . . You can list more theoretically feasible methods, but don't they all have the problem that far water can't quench near thirst?
In fact, there is a way, and. . . . . . . . . . . . . .
It's about broccoli again
However, Broccoli Jun swore to God that it was definitely not sulforaphane this time.
Here's the thing. 20 19 May, this time last year, Science published a striking paper. A team at Beth israel Deaconess Medical Center at Harvard University found a way to increase PTEN protein, starting with those things that control PTEN protein in cells. The compounds used are quite rich in cruciferous plants such as broccoli, and even sold as health products!
As mentioned above, PTEN has a great function, so it must be specially supervised by cells. Many protein will monitor it and tell it to work only when it needs to put on the brakes. One kind of protein, called WWP 1, will put a mark on PTEN to tell it that the brake will not work for the time being.
Obviously, if we try to kill WWP 1, can't we release some or even many idle PTEN proteins?
And they found that something that can effectively kill WWP 1 is far away, near the dining table, which is indole -3- methanol in broccoli. It looks like this:
So does this indole -3- methanol have a therapeutic effect on patients with PTEN gene mutation? There should be no clear research evidence at present, but I believe many people can't wait to try. After all, there is no other good way. Indole -3- methanol is an easily available health care product.
Back to the original topic, about EP300 gene, there are various other autism genes. Broccoli Jun believes that he will eventually start with the PTEN gene research method mentioned above, and either modify the gene or add the correct gene, or try to increase the amount of normal protein, or adjust what interacts with this protein. Make every effort to increase the number of protein who can work normally. However, the biggest obstacle should be the source of evil, money.