The new success of gene therapy

Gene therapy coped with Fabry's disease in a week

Svetlana Maslova, Hi-tech+

Currently, patients with this rare chronic disease have to visit the hospital every two weeks to perform injections that restore the deficient level of enzymes. In Canada, several patients have undergone an experimental course of treatment with gene therapy and have not been dependent on regular visits to medical institutions for several years.

Article by Khan et al. Lentivirus-mediated gene therapy for Fabry disease is published in the journal Nature Communications – VM.

For the first time, gene therapy was used to treat people with Fabry 's disease in 2017. To date, treatment has already been provided to five men who have fully or partially restored the levels of deficient enzymes to normal in just one course.

Today, patients with Fabry's disease are shown regular therapy: intravenous enzyme infusions are performed every two weeks to maintain the functions of internal organs. Without treatment, the disease affects the heart, kidneys and brain and leads to various health problems. In men, the disease is most severe. Canadian scientists from The University of Calgary sought to develop a treatment that would permanently or even permanently restore the levels of defective enzymes to normal.

To achieve this result, they collected a small number of stem cells from the patients' blood, and then, using a viral vector, injected copies of a fully functional gene into them, which is responsible for the production of the necessary enzymes. After these manipulations, the modified cells were injected back into the patients.

As a result of the treatment, all five patients began to produce deficient enzymes at a level close to normal in just one week. At the moment, three men have been in a stable condition for several years now.

"We see that just one course of treatment is enough to benefit patients. Now we need to estimate how long the effect will last," the authors commented.

As part of this stage of research, scientists observed patients from 2017 to 2020. The final assessment of their condition is planned to be completed by 2024. This long period should demonstrate whether the volunteers need to be reintroduced cells or their stable condition remains unchanged.

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