"Hand of God" builds a gene therapy bridge
Dangdang! Knock on the blackboard! ! look here! ! !
In 2016, the National Natural Science Foundation has been on the list for some time! Are the teachers still immersed in joy and unable to extricate themselves? O(∩_∩)O is awake, our goal is: the fund is there every year, more in the next year!
Every year, the National Natural Science Foundation is the vane of China's scientific research field! By analyzing the nature of the country, we can see the latest research hotspots, the subjects that the state attaches to the current state, the problems that need to be solved in the clinic, the signals of the direction of the national research investment, etc., so as to guide future research ideas, let us apply for other topics and The success rate of the article published is higher!
Today, Zhang Bo led everyone to analyze one of the national hottest words - CRISPR/Cas9 (funded projects: 81; funding amount: about 32 million) , I hope that our analysis will let everyone have the latest application of this technology. Deeper understanding!
Known as the "Hand of God", the CRISPR/Cas9 technology uses sgRNA recognition genes to mediate cas9 enzyme localization and cleavage of genes. When a homologous recombination template is added at the same time, the effect of gene editing can be achieved. Although this is not the first tool scientists use to edit DNA, it is more accurate, easier to operate, and cheaper than previous technologies.
(Image from the network)
Since 2014, with the maturity of cas9 technology, its application has become more and more extensive. One of the most important applications is the use of CRISPR/Cas9 to construct transgenic model animals (mainly genetically edited mice) to provide pre-clinical basic research on animal-level treatment. In the clinical research, the basic experiments of cell experiments have been done more and more, and if there is no obvious effect in vivo or in vivo experiments, the significance of scientific research will be greatly reduced. The same experiment, combined with animal experiments, is far more convincing than the cell level; the same article, together with the data from animal experiments, is a complete Story. If not, even the editors of CNS can help. The transgenic mice just made up for this short board. Different from the tumor formation of nude mice, transgenic mice can better simulate tumor models through genetic modification, which can minimize the occurrence and development of human cancer.
It can be said that transgenic mice are the bridge between the gene therapy and the clinical experiment.
(Image from the network)
A considerable number of diseases are expected to be based on CRISPR/Cas9 technology, verified by genetically edited animal experiments, and then applied to human clinical treatment. For example, at the end of June 2016, the National Reproductive Advisory Committee of the National Institutes of Health (NIH) passed a resolution to approve the CRISPR gene therapy program for human clinical testing. On July 21, 2016, Nature's homepage news report that China's Sichuan University Huaxi Hospital is expected to take the lead in conducting the world's first CRISPR/Cas9 gene editing technology to treat non-small cell lung cancer.
In a Cell Research article published on August 30, researchers first detected the H530R mutation in AMPKg2 from diseased families, and used gene editing techniques to construct transgenic mice expressing the mutant gene and gene knock-in. Mouse model . Both mouse models successfully reproduced the patient's main pathological features, which confirmed for the first time that this mutation led to the occurrence of PRKAG2 heart syndrome. The researchers then loaded the gene editing tool CRISPR/Cas9 in a safe and efficient AAV9 (adeno-associated virus) and delivered it to mouse heart expression , enabling specific editing of the mutated alleles, successfully correcting and repairing the mouse heart. Form and function. And significantly reversed the main indicators of the heart disease. It was first confirmed that the CRISPR/Cas9 gene therapy strategy can be applied to human dominant genetic heart disease. This clever two-way use of CRISPR/Cas9 gene editing technology for clinically relevant research is increasingly receiving attention from mainstream impurities.
These news are important signals for the industrialization of CRISPR technology, and major investment institutions continue to be optimistic about the huge prospects of CRISPR technology to overcome the refractory diseases such as genetic defects, cancer and AIDS.
So what are the advantages of Cas9 in building transgenic mice?
1.CRISPR/Cas9 constructs transgenic mice with shorter cycle
Using traditional ES targeting techniques, it takes about 10 months from vector construction to F1 heterozygotes. With the Cas9 technology, since only mRNAs are injected into mouse embryos, the step of screening ES cells can be omitted, which takes only 4-6 months.
2. Since Cas9 cleavage increases the efficiency of homologous recombination and reduces the process of screening ES cells, the cost of constructing transgenic mice with CRISPR/Cas9 is lower.
3.CRISPR/Cas9 can construct multiple mutations at the same time
As long as a variety of sgRNAs targeting Cas9 are available, this technique can simultaneously create multiple mutations in a single fertilized egg, saving significant time costs.
4.CRISPR/Cas9 can build animal models of any species
The Cas9 technology is not limited to mice and rats. It can theoretically construct mutations of any species. The current experiments include pigs, sheep, monkeys and so on.
It can be seen that on the one hand, along with the results of the National Natural Science Foundation, the continued popularity of gene editing is predictable, and future research investment is inevitable; on the other hand, animal models accompanying gene editing are becoming more and more accessible. The more common it is, the more demanding animal experiments in articles or funds will be!
Don't let high-scoring articles and fund winning bids miss us because of the lack of animal experiments!
More on the introduction of animal experiments, article analysis, technical exchanges, welcome to poke Zhang Bo small mailbox!
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