Currently, repairing the stem cells involves removing them from their hiding places in the deepest part of the body, then genetically altering them and returning them to the patient.
However, new research conducted at Harvard University has proven that they can do it in the patient's own body (at the moment, in mice).
We owe our long lives to stem cells, which are nested inside certain body tissues and constantly replace old cells. In recent years, scientists have been able to correct genetic diseases by eliminating these stem cells, editing their genomes and then re-implanting them in the patient.
The standard procedure for editing stem cells, removing them from the body, however it brings many complications: Because stem cells can die in the culture dish, the patient's immune system can reject them once they have been transplanted or simply cannot be activated again.
Now, new research led by Harvard scientists has successfully edited the stem cell genes while still in the body. Based on previous work, the team loaded the gene editing machinery into different types of adeno-associated viruses (AAV). These viruses can penetrate the cells of mammals, and have been altered so as not to cause disease and to deliver a payload of gene editing machinery.
In the tests in mice, the researchers used the AAVs to obtain the CRISPR gene editing system in different types of skin, blood and muscle cells and progenitor cells. Up to 60 percent of the stem cells in the skeletal muscle were edited, as well as up to 27 percent of the skin progenitor cells and 38 percent of the stem cells in the bone marrow.
The team says that this breakthrough could lead to new treatments for genetic diseases, particularly those such as muscular dystrophy that depends on tissue regeneration. As he explains Sharif Tabebordbar, lead author of the study:
Our study demonstrates that we can permanently modify the genome of the stem cells and, therefore, their progeny, in their normal anatomical niche. There is great potential to advance this approach and develop more durable therapies for different forms of genetic diseases.