A breakthrough in genetics

Stem cells have learned to edit inside the body

Georgy Golovanov, Hi-tech+

A group of scientists from Harvard has successfully made changes to the genes of stem cells without removing them from the body. This technology will lead to the emergence of new drugs against genetic diseases, especially those that interfere with tissue regeneration.

The treatment of genetic diseases is similar to the purification of water in a river: if you collect garbage only in the lower reaches, it will not become cleaner — you need to solve the problem at the source. Similarly, the treatment of diseased cells will not help if you ignore the stem cells, which will quickly replace healthy cells with affected ones.

Now stem cell therapy requires first removing them from the body, then genetically modifying them and putting them back, says Harvard Gazette (Editing genes at the source).

This complex procedure has many risk factors: stem cells may die in vitro, the patient's immune system may reject them after transplantation, or they may simply not work as planned.

Scientists from Harvard suggests simplifying the procedure — and increasing its effectiveness.

"When you extract stem cells from the body, you take them away from a very complex environment that nourishes and supports them, and they experience something like shock," said Amy Wagers, the head of the study. — Isolation of cells changes them. Transplantation changes them. Genetic changes without all this would preserve the regulatory interactions of cells — that's what we wanted to do."

How it works

Scientists have loaded the CRISPR gene editing mechanism into various types of adeno-associated viruses (AAV) that can penetrate mammalian cells without harm to them. In an experiment on mice, scientists delivered CRISPR to stem cells of skin, blood and muscles. To understand whether this technology worked, stem cells were instructed to activate fluorescent genes.

The method worked. Scientists found that 60% of the stem cells in the muscles, 38% in the bone marrow and 27% in the skin turned red.

A drawing from an article by Goldstein et al. In Situ Modification of Tissue Stem and Progenitor Cell Genomes, published in the journal Cell Reports – VM.

In addition, other skin cells were also edited, which indicates the transmission of changes to offspring.

The study shows the possibility of permanently modifying the genome of stem cells and, as a result, their offspring, in their usual anatomical niche. This approach has great potential in developing more reliable therapies for various forms of genetic diseases.

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