Recently, the U.S. Food and Drug Administration (FDA) approved the marketing authorization of CRISPR Cas9 gene editing for sickle cell disease, a blood disease that originally had no good method[1].
The world's first CRISPR gene editing**As the world's first CRISPR gene editing**, Casgevy enrolled 29 of 31 evaluable patients in a global clinical trial with 29 of 31 evaluable efficacy patients (93.)5%) achieved at least 12 months without episodes of severe vaso-occlusive crisis (VOC). Not only that, gene-edited cells can survive and function continuously in the patient's body for a long time. At the 3rd International Summit on Human Genome Editing in March this year, the first sickle cell disease anemia patient to receive this ** came forward to share her ** experience, she said that since receiving the new **, there is no longer a need for continuous blood transfusion**, and the unbearable severe pain has disappeared [2]. 
The patient appeared at the conference to say that sickle cell disease is a hereditary disease, because of the genetic defect carried by the patient, the red blood cells that should be round mutate into sickle-shaped cells, this "little sickle" can not carry oxygen well, and it is hard, it is easy to block blood vessels, causing hypoxia, anemia and many other problems, and patients are very easy to feel fatigue and weakness. In addition, patients may also have complications such as severe pain, infection, and organ damage due to the disease, and the risk of cardiovascular diseases such as stroke is also high, and the life expectancy is seriously affected. Sickle cell disease for a long timeThere is no cure, pain relievers and blood transfusions** maintenance are commonplace. Previously, there was only one targeted method for this disease, and that was bone marrow transplantation, but the risk was high, and even after transplantation, most patients could not, and it was not easy to find a donor and. The advent of Casgevy, the first CRISPR gene editing in humans, has completely upended the rules of the game for this intractable disease. 
*: Photo.comBy injecting a large number of gene-edited bone marrow cells into the patient's body, these modified cells will continue to survive and function in the patient's body, which can free the patient from long-term blood transfusions and relieve complications such as severe pain and fatigue. This kind"Once, long-term".It is expected that sickle cell disease, which has no good methods, will become a chronic disease that can be well controlled and greatly improve the quality of life of patients"Genetic Scissors" opens the door to a new world
CRISPR, a cutting-edge gene editing technology, also known as "gene scissors", won the Nobel Prize in Chemistry in 2020. Its potential goes far beyond sickle cell disease. Theoretically, as long as the disease-causing gene problem can be found, this "sharp scissors" may be carried out by cutting and modifying the DNA sequence**. 
The approval of CASGEVY is actually the first application of CRISPR, a Nobel Prize-winning achievement, which proves that this cutting-edge technology has broad potential for genetic diseases. In February last year, the scientific journal Nature Reviews Cancer published an article titled CRISPR in Cancer Biology and Therapy, which took stock of the progress of CRISPR in cancer research. 
For example, using the convenience of the CRISPR system, researchers can quickly knock out certain genes in cell samples or organoid samples in the laboratory to see how this knockout affects cancer cell growth or cancer cell response to drugs. This will dramatically increase the speed of research in many areas of cancer – meaning that more research is likely to emerge at the same time, and the final quantitative change will drive the qualitative change, allowing some excellent anti-cancer** to emerge sooner. In addition, in the current "immunity" of cancer fever, the potential of CRISPR is also very huge. In the "1.2 million shots" of CAR-T** that people may have heard of, the use of CRISPR to knock out certain antigens can reduce the risk of common immune rejection and graft-versus-host disease, making it safer;CRISPR technology can also help generate more potent CAR-T cells and improve anti-cancer effects. 
The CAR-T reinfusion process (Shengnuo patient***, for example, tumor-infiltrating lymphocytes** (TIL**), can also be improved with the help of CRISPR gene editing. The cutting-edge TIL**KSQ-001EX, which was tested in Phase 1 and Phase 2 clinical trials, was powered by CRISPR technology. The basic principle of TIL** is to extract strong T cells from cancer patients that can detect and kill tumor cells, and then expand them in large quantities in the laboratory, and then infuse them back into the patient's body to exert their effects. Researchers have found that CRISPR technology can be used to gene-edit extracted T cells to inhibit a gene called SOCS1, and then these modified T cells will have stronger anti-cancer power and more long-lasting effects. At present, the study is being carried out in a variety of solid tumors such as melanoma, head and neck cancer, and lung cancer. On January 4 last year, researchers from Harvard Medical School in the United States published an article in the sub-journal of the journal Science, introducing a shocking new cancer vaccineUse cancer cells to make vaccines to fight cancer
The basic principle of the network is to use the potential of living tumor cells to locate and target tumors, and transform tumor cells through CRISPR technology, so that they have the dual ability to kill tumors and activate the immune system, killing the original cancer cells at the same time, but also allowing the immune system to find and remember cancer cells, and play a role in prevention. These ** cancer cells are known as THTC, and in preclinical studies, they have successfully eliminated advanced glioblastoma tumors in mouse models. In order to ensure safety, the researchers also installed multiple "safety switches" on these modified cells, which can be activated when needed to eliminate these modified cancer cells. Today, CRISPR technology has opened a whole new door to human medicine. By cutting and modifying DNA sequences, many incurable and incurable diseases that were originally unavailable and incurable will have a new glimmer of hope. It is believed that after the launch of Casgevy, more and more cutting-edge new drugs and new products brought by CRISPR gene editing technology will appear, changing the fate of patients!
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