Our longevity we owe to the stem cells that reside deep within certain tissues of our body and replace old cells. In recent years science has made significant progress in the treatment of some genetic diseases by editing the stem cell genome. To do this, they first remove from the body, edit, making desired changes in their genome, and then placed back into the patient’s body. But this procedure carries a number of difficulties. Scientists from Harvard University reported that everything is in a short time can be avoided. The researchers conducted the first successful gene editing of stem cells directly inside the body. The results of their work reported in the journal Cell Reports.
Edit genome stem cells is a complex process
The process of treating genetic diseases can be compared to trying to clean the river from garbage. If you just collect all the debris on the river bottom downstream, then eventually the river will become dirty, if that does not solve the problem with garbage upstream. Similarly, the ineffective treatment of diseased cells. It will not help if only not to resort to the use of stem cells that can quickly replace damaged or old cells with new and healthy.
Currently, the editing process of stem cells involves their extraction from the body, further genetic changes and eventually reverse the integration of the edited cells back into the body. At all stages of this very complex procedure has a high risk of errors and failures: stem cells can simply die inside the Cup for cultivation, the patient’s immune system can begin the process of rejection of the edited stem cells after transplantation, or the new cells will not have any positive actions.
“When you extract stem cells from the body, you extract them from very complex environment that nourishes and supports them. The extraction creates a real shock for them,” explains Amy Wagers, leading specialist of the study.
“Isolation of stem cells alters their. Transplantation modifies them. The possibility of making genetic changes in their genome without much additional interaction with them is a key task of our research.”
Building on previous work on gene editing of stem cells, the Harvard team of scientists has used AAV viruses (AAV) to deliver the necessary editing of the genome of the substances inside of cells. These viruses do not cause disease in humans and, therefore, only cause a very mild immune response.
For method validation, the researchers used laboratory mice. For the introduction of AAV-viruses in different types of rodent cells (skin, blood, muscle, and progenitor cells (refer to the initial stage of cell development)) the researchers used a gene editing technology called CRISPR. For the possibility of visual observation of result, stem cells also activated reporterne genes that are highlighted with a fluorescent red color.
Scientists expected success. Researchers report that up to 60 percent of the stem cells of skeletal muscle, up to 27 percent of skin stem cells and to 38 percent of the stem cells of the bone tissue of mice was highlighted in red, indicating that they were successfully edited. Following this, scientists add that other skin cells also were subsequently changed, which speaks in favor of the transmission of genetic information from the edited stem cells to other cell types.
A team of Harvard scientists reported that their research breakthrough could lead to the development of new treatments for genetic diseases, particularly such as muscular dystrophy, which is associated with the problem of tissue repair.
“Until now, the concept of delivering healthy genes into the stem cells using AAV viruses were not practical, because these cells, being in the living body, so quickly share that the delivered genes are rapidly dissolved from them,” says Sharif of Tableborder.
“Our study demonstrates the ability to continuously edit genome stem cells and their daughter cells in their natural environment. This opens a large potential for further improvements of this method of gene editing, and developing on its basis a more reliable and effective methods of treatment of various forms of genetic disease”.
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