Innovative Strategies Being Developed to Ensure the Clinical Application of CRISPR-Cas9 Gene Editing System

 

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Currently, the CRISPR-Cas9 gene editing system has produced exciting results in the field of gene therapy, which has inspired scientists to take it in the appllication of treatment for human hereditary diseases. Recently, a research report published in the international journal Nature Medicine, scientists from Germany have studied the human body's immune response to CRISPR-Cas9 and found that the human body can produce a wide range of immunity to Cas9 protein. Researchers are now developing a variety of innovative strategies to ensure that the CRISPR-Cas9 gene editing system is safe to use in a range of clinical applications.

In this study, the researchers analyzed the potential benefits and potential risks of CRISPR-Cas9. CRISPR-Cas9, which is a new molecular genetic editing tool that helps scientists make specific changes to the DNA of human organisms, animals and plant organisms. This technique is similar to a special molecular scissors that help scientists cut, silence or remove specific DNA sequences, and it can be used to modify cells in vivo or in vitro, or be transported directly into the body. At present, researchers have demonstrated the effectiveness and usability of this gene editing tool in animal models.

However, until so far, there is not enough evidence to assess the potential risks and benefits of using this technology in human organisms. Researchers may be able to successfully fill the gap in this field of research by studying the immune response of the human body to the CRISPR-Cas9 system. Researcher Schmuck-Henneresse said that the Cas9 protein is derived from the genus Streptococcus, which forms a key part of the CRISPR-Cas9 system. Streptococcus infection is very common in the human body, so the researchers speculate whether the human body pre-existing immunological memory of Cas9 protein.

Researchers can find that T cells in the body respond to Cas protein in almost all healthy human subjects. Cas molecules are derived from other bacteria, such as streptococci and gastrointestinal bacteria, which can produce special types of immune response, and the occurrence of this phenomenon may be due to the high similarity between enzymes. During the treatment of gene therapy, these immune cells can produce some bad immune reactions, which potentially affect the safety and effectiveness of the CRISPR-Cas9 gene editing technology. Obviously, there may be some risks in the CRISPR-Cas9 technology, this is what the researchers need to consider before the application.

According to the researchers, this means that cells that are genetically modified in the body should not be imported into the patient's body where the CRISPR-Cas9 system remains active. Researchers have now developed a detection technique to ensure that cell products can be safely used, which can reliably determine whether the risk of immune response in the patient's body is at a lower level. However, some hereditary diseases produce tissue defects that cannot be modified outside of humans, so researchers need to find new ways to suppress the dangerous immune response of the CRISPR-Cas9 gene editing tool.

In this study, researchers have developed a new technology that ensures the safety based on the use of the CRISPR-Cas9 gene editing tool, which works by isolating and expanding regulatory T cells to control the body's "disgusting" immune response. Initially, the researchers conducted corresponding clinical trials in the body of patients undergoing kidney transplantation or bone marrow transplantation, which have achieved positive results.

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