CRISPR: Panacea for fatal diseases or phantom perspective

Author : Nina Hlushchenko

Source : 112 Ukraine

This method is expected to help in the treatment of cancer and AIDS, coping with congenital genetic diseases and even raising crop yields in the fields
09:57, 30 January 2018

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The Intramural Research Program

Clustered Regularly Interspaced Short Palindromic Repeats (it will be more correct to say CRISPR/Cas9, but we will just use CRISPR in this article) is a method that allows you to edit a person's genetic code, removing unwanted genes or introducing new sequences. With its help, you might turn on or off some processes in the body. Over the next six years, the US government plans to invest $ 190 million in research related to gene editing, including CRISPR. The authorities hope to introduce a new treatment method in the whole country. Currently, we are talking about editing the non-productive human cells.

CRISPR would be used as part of therapy in the fight against cancer and genetic diseases. Just recently, scientists have found that activation of one gene by means of CRISPR allows getting stem cells from of connective tissue cells, which is a promising material for treating a great number of diseases.

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According to BusinessInsider, CRISPR will radically change agriculture in the nearest 10 years. The method will make cultures more resistant to drought and other challenges. Experts believe that the first plants grown by this new method of genome editing would appear on the supermarket shelves in a couple of years. Now China is the leader in researching this area: it is ahead of the US by the number of publications on this topic and is already conducting experiments in the field of animal husbandry. Thus, cows resistant to tuberculosis, and pigs with a reduced content of white fat were created. China has a great motivation to succeed in this direction. A country with such a huge population understands that someday a problem of the lack of food might be faced.

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If CRISPR is so effective, then why doesn’t the world use it everywhere? Today China has become the most active country in terms of clinical trials of the method in humans. According to The Wall Street Journal, the cornerstone was the question of ethics. In the summer of 2016, the United States allowed testing of technology on humans, but no serious success in this direction has been heard. Although there is no formal ban, American doctors have to report more severely about their work and observe a huge number of rules. China does not have such formalities, so doctors are more willing to experiment with CRISPR.

Now CRISPR is most actively considered as a tool to fight cancer. The first study on a living person has been conducted just over a year ago at Sichuan University. Scientists have introduced modified T-lymphocytes to a patient with lung cancer in order to edit a gene that controls the expression of the PD-1 protein. If scientists can “turn it off,” it will help the body to fight the tumor.

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The first such experiment in the United States will begin in March 2018. Scientists from the University of Pennsylvania will conduct research on 18 volunteers, patients with multiple myeloma, sarcoma, and melanoma. In addition to removing the gene responsible for the creation of the PD-1 molecule, the receptor responsible for "alerting" the immune system will be removed. Instead, patients will receive a genetically modified receptor that will direct cells straight to the tumor.

At the end of the year, the first clinical trials in Europe are planned. The doctors will treat a patient with a hereditary blood disease - beta-thalassemia.

In all cases, the modification of cells occurs in the laboratory, and only after they will be implanted in the body of a living person. Scientists suppose that in the future it will be possible to even fight with cancer preventively - editing the genome of the embryo, removing from it genes that provoke a dangerous disease. However, the situation is complicated by the fact that heredity is not the main reason for different types of cancer.

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It is planned to fight other inherited diseases or genetic abnormalities in the same way. Last year, the United Kingdom allowed editing the genome of a human embryo, although only for fundamental purposes with the obligation to destroy it within 14 days. Chinese scientists in June 2016 conducted the first experiment on editing the genome of the embryo, but it turned out to be unsuccessful. According to Meduza, probably the reason is that the ovum has got edited when the spermatozoon was already inside and the copying of the parental genes began. The more copies are created, the more difficult it is to edit the mutation. However, after that, in August 2017, a successful experiment on editing the genome of the embryo was conducted by American and South Korean scientists. They managed to take out the embryo of hypertrophic cardiomyopathy.

In addition to the scientific questions, this touches some ethical aspects too. If mankind learns to change genes to get rid of diseases, then it will be possible to use this opportunity for other purposes. For example, to combat baldness, to make the body prone to sporting successes or to determine the appearance of a person. Would it be impossible to find the line between medical needs and the desire to obtain super-abilities for your children? The National Academy of Sciences of the United States (NAS) bears alarm and suggests that scientists and doctors around the world agree on the use of CRISPR only for therapeutic purposes. But where is the guarantee that such operations, even if they are banned officially, will not be conducted illegally?

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Stanford scientists have conducted a blood test for the presence of antibodies to two types of Cas9 protein and found that about 65% of the subjects are carriers of T cells that protect them from the effects of these proteins (22 of them were newborns and 12 adults). That is, they have immunity against gene therapy, and it might not work for them or even lead to some toxic consequences. The problem is that CRISPR experiments use proteins obtained from two kinds of bacteria - pyogenic streptococcus and Staphylococcus aureus, which are regularly found in human life. The solution might be the use of bacteria, with which a person has not faced yet. But this would not be used in the nearest future.

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In addition, scientists have not gone so far in the study of genes and possible consequences of genome editing. With the current level of understanding, we can find ourselves in an unpleasant situation, when, without proper studying the effect of radiation on the body, people enthusiastically used the radioactive cosmetics without realizing that it was extremely dangerous and dreadful.

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