Geneticists have found a way to combat a rare inherited disease SciImmunol: gene editing can normalize immune response

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German scientists from the Center for Molecular Medicine named it after him. Max Delbrück in Berlin has developed a method to combat some genetic diseases that cause an excessive immune response. It is based on gene editing using the CRISPR-Cas9 tool. The study was published in the scientific journal magazine Science Immunology (SciImmunol).

Geneticists have turned their attention to familial hemophagocytic lymphohistiocytosis (FHL), a rare and dangerous immune system disease that usually occurs in infants and children younger than 18 months. It is caused by various gene mutations that prevent the normal functioning of cytotoxic T cells, or T lymphocytes, which are immune cells that destroy tissues infected by viruses.

If a child with FHF is infected with certain viruses, their T cells may not be able to fight the infection. Instead, the immune system triggers a cytokine storm, an inflammatory response that affects the entire body.

Today, FHF is treated with a combination of chemotherapy, bone marrow transplant, and immunosuppressive drugs, but the mortality rate remains high.
The team was able to repair the damaged gene called perforin, which is often defective in FHF patients. During experiments, laboratory mice with artificially induced SHF were injected with corrected perforin, and the rodents subsequently recovered.

Researchers hope that similar treatment will help people, normalizing their immunity for a long time, or even forever.

Previous scientists opened A new way to treat bacterial infections using viruses.

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