Immunodeficiency gene therapy was tested on mice
"Genetic scissors" cured severe immunodeficiency
A large international group of scientists has once again confirmed the benefits of the CRISPR-Cas9 system, or as it is also called "genetic scissors", reports Infox.
With the help of a "genetic operation", the authors were able to edit the genome of cells of patients with severe immunodeficiency – chronic granulomatosis caused by genetic breakdowns. This disease is practically untreatable, and a person is forced to take antibiotics all his life. But the main danger for such patients is life–threatening infections.
The disease causes a defect in the NOX2 protein, which is one of the main agents of the immune system.
In their study, the authors took damaged blood stem cells from patients with granulomatosis and restored the function of genes in NOX2. Then these cells were implanted into the body of mice with impaired immunity. After implantation, the cells lived in the mice for five months, and during this time the immune system of the animals was restored. This suggests that the method works in this case, the authors say. Although they add that all these manipulations are associated with great risks. Therefore, in clinical practice, the method based on the CRISPR-Cas9 system will not appear soon. Not earlier than in 10-15 years.
The authors report about their research in the latest issue of the prestigious scientific journal Science Translational Medicine (De Ravin et al., CRISPR-Cas9 gene repair of hematopoietic stem cells from patients with X-linked chronic granulomatous disease).
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12.01.2017