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Researchers at Harvard University have shown that gene editing systems can be delivered directly to the place of residence of cells rather than being used in a petri dish. The researchers believe the findings have important implications for biotechnology research and the development of treatments for genetic diseases. Researcher Amy Wagers says that to correct genetic mutations that cause disease, the relevant stem cells must be changed.
If these cells are not modified, corrections made in other cells are eventually replaced by diseased cells. The attachment of the stem cells causes the replication of healthy cells that will eventually replace the diseased cells. Currently, stem cells must be extracted, kept alive, genetically modified and then reintroduced into the body of the person.
This process disrupts the cells and they can be rejected or not transplanted into the person. She says that making changes without removing the cells would preserve the regulatory interactions of the cells. The betting team used an adeno-badociated virus (AAV) that infects human and mouse cells, but does not cause disease, as a means of transport.
The team designed an AAV-engineered gene export cargo that would be transmitted to different types of stem cells from skin, blood and muscle, as well as progenitor cells. To check the functioning of their cells, the team used mice and activated a "reporter" gene using the method that makes them bright, red fluorescent when the reporter gene is activated.
The team discovered that the new method was 60% effective with 60% of stem cells becoming red in skeletal muscle. Up to 27% of the cells responsible for different types of skin became red and 38% of the bone marrow stem cells became red. The team notes that even a single healthy stem cell could be enough to stop a defect.
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