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For the first time, scientists have succeeded in genetically modifying stem cells in the body of mice, which could potentially open new perspectives for stem cell therapy.
Stem cells can turn into all kinds of other cells and the body uses these various tools for growth and repair. Due to their remarkable abilities, the use of stem cells in some medical treatments is attracting interest, but this path is not always easy.
For example, in a particular type of bone marrow transplant, the stem cells that produce the blood must be removed from the body and genetically modified before being transfused to the patient.
The results of this new study in mice indicate that we could possibly remove the stage of complicated and risky extraction: the necessary genetic modifications could be performed. in vivowhich will probably be both faster and more efficient than the techniques used today.
"When you extract stem cells from your body, you extract them from the very complex environment that feeds and maintains them, and they are sort of shocked," said the principal investigator for the body. study, Amy Wagers, of Harvard University of Mbadachusetts. .
"Isolating the cells changes them.Cell transplantation changes them.Getting genetic modifications without doing this would preserve the regulatory interactions of the cells – that's what we wanted to do."
And researchers have done so using what's called an adeno-badociated virus (AAV) – a virus able to enter the body to infect and alter human cells (or, in this case, the mice) without causing disease.
In tests on mice, AAVs with CRISPR gene editing technology were administered to different types of stem cells from skin, blood and muscle and progenitor cells (similar to stem cells but more specific and developed).
Through the use of activated "reporter" genes that shine from a fluorescent red color inside cells, researchers have been able to observe genetic modifications: up to 60% of cells strains in skeletal muscle, up to 38% of stem cells. in the bone marrow and up to 27% of the progenitor cells of the skin.
These figures may not seem so high, but they are a very good start. The scientists then found evidence of the changes applied by stem cells propagating in the bodies of animals.
"Until now, the concept of healthy gene transfer to stem cells with the help of AAV was not practical because these cells divide so rapidly in living systems." The genes thus delivered will be rapidly diluted cells, "says one of the team members, Sharif Tabebordbar of the Broad Institute in Mbadachusetts.
"Our study demonstrates that we can continuously modify the genome of stem cells, and hence their descendants, in their normal anatomical niche, and this approach could be used to develop more sustainable treatments for different forms of genetic diseases."
There is still a lot of work to be done, especially to demonstrate that the same techniques can work in humans and in mice, but this new study shows that the AAV and CRISPR approach is one of the our most promising.
In addition to providing us with more effective stem cell tools for fighting disease-based diseases, the research also opens up different options for studying genetically engineered stem cells in their natural habitat rather than in the laboratory.
"The approach we have developed solves all the problems you pose by extracting stem cells from a body and allows you to fix a genome permanently," says Wagers.
"AAVs are already used clinically for gene therapy, so things could begin to evolve very quickly in this area."
The search was published in Cell reports.
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