For the first time, Chinese scientists managed to create directed mutations in monkey embryos. This technology will help find cures for many diseases. It is also possible that in the future, gene modifications will guarantee the birth of absolutely healthy people.
Thanks to the success of Chinese scientists, the potential for precision gene editing is finally starting to be realized. In the short term, this will allow the creation of primates with human diseases and testing new treatment methods on them. Moreover, in the distant future, genetic modification of human embryos may be approved, which will save humanity from congenital diseases and unhealthy predispositions.
Scientists from the Model Animal Research Center of Nanjing University presented the first twin monkeys with a targeted mutation to the scientific community. Previously, such directed mutations could only be carried out in mice, now it has been possible in primates too - using the promising CRISPR / Cas9 technology developed at the Massachusetts Institute of Technology. It uses customizable RNA fragments to control DNA cutting and insert the desired modified fragment into the genome.
Transgenic mice have long been used as models for the study of human disease. It is a convenient model organism: mice reproduce rapidly and the desired rare mutations can be obtained using a fairly simple homologous recombination method. However, the low rate of homologous recombination makes it unsuitable for monkeys that reproduce slowly.
Attempts have been made to genetically modify primates with programmed viruses that create mutations very efficiently, but in unpredictable locations and uncontrolled numbers. However, CRISPR / Cas9 technology overcomes these problems.
In experiments on monkeys, scientists were able to modify three target genes with a 10-25% success rate. The scientists then replaced three target genes in more than 180 embryos. After that, 83 genetically modified embryos were implanted in the monkeys, and one of the 10 pregnant monkeys has already given birth to twins with mutations of two genes: Ppar-γ (regulates metabolism) and RAG1 (participates in the immune system).
The combined mutations in PPAR-γ and RAG1 are not disease specific syndrome, although each of these genes is associated with different diseases. So far, scientists have not yet fully analyzed the condition of the monkeys and must determine whether the mutation has occurred in all animal cells.
Scientists are currently working to improve the efficiency of genetic modification of monkeys, since the current laborious process gives a minimum percentage of success. Also, scientists are studying old attempts to instill human diseases such as autism and immunodeficiency in monkeys. Chinese researchers will try to replicate these experiments using the more promising CRISPR / Cas9 technology. If successful, there will be new opportunities to study and treat a variety of human diseases.
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