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Aurora magazine

The first attempt at genetic editing was done in vivo

In the future, will it be possible to correct a genetic error directly in the body? Maybe yes. The scientists at the Oakland Benioff Hospital have applied gene therapy to a man with Hunter's disease. If all goes well, the body will incorporate the correct version of the defective gene, hence a regression of the disease.

In standard gene therapy, offensive viruses are used to replace diseased genes with their correct versions. Usually, scientists apply the procedure on a cell culture of the patient, to be re-planted at a later time. In this case, instead, they used genetic editing directly in the patient's body.

Scientists have operated a man suffering from a disease called mucopolysacaridosis type 2, or Hunter's disease. Those who suffer from it accumulate sugars in the organelles of the cells, all because of a chronic scarcity of the enzyme Ids. In the long term, the accumulations of sugar cause problems of sight, hearing, heart. To date, the only available treatment is weekly enzyme infusion, but gene therapy may make it unnecessary.

Phases 1 and 2 of the experiment are intended to verify the safety of the treatment. Only at this point will you go to Stage 3, which will serve to verify its effectiveness. For this purpose, researchers have already recruited 9 other patients with the disease who will undergo short-term surgery. In addition, similar experiments are ongoing for type B hemophilia and type 1 mucopolysaccharides, or Hurler-Scheie syndrome. The criterion used is always the same.