Researchers have been successful in genetic modification of stem cells inside the body of mice for the first time ever. This feat can unlock various avenues in stem cell therapy, which have not been explored so far. The study has been published in Cell Reports.
Stem cells can grow into all other types of body cells and the body uses this ability for its growth and repair in several parts. Because of the potential in stem cells, researchers are always looking to incorporate stem cells in the medical treatments but it has not been easy so far. For example in the case of a bone marrow transplant, the stem cells which produce blood have to be first removed from the human body, genetically modified and only then be transfused back into the human body.
From the results of this experiment on mice, it is likely that the complicated extraction step can be bypassed. The genetic edits which are needed can be performed in vivo, which is estimated to be much faster and effective than the techniques which are currently used.
Amy Wagers from Harvard University who is the principal researcher behind the study commented that when stem cells are taken out of the body, they are being removed from the circumstances which provide nourishment and helps to sustain them. As a result of this change, they go into shock. The researchers wanted to make the genetic changes in the cells without isolating or transplanting them as it changes them altogether.
Researchers did this by using adeno-associated virus(AAV). This virus is able to enter the body for infecting and altering the cells without generating any sort of disease. In the tests which were conducted on mice, AAVs packed with CRISPR gene editing technology were released in various types of skin, blood, muscle stem cells, progenitor cells.
With the help of activated reporter genes which turn to fluorescent red inside cells, researchers could observe the genetic changes, upto 60 percent of stem cells in skeletal muscles, 38 percent in bone marrow and 27 percent of progenitor cells.
Sharif Tabebordbar from Broad Institute, Massachusetts who is a member of the team said that till now delivering genes to stem cells using AAV was not possible as the cells get divided very fast in living bodies. But the team was successful in modifying the genome of the stem cells within the body itself.
However, the job is not yet done as this has to be successfully demonstrated in the human bodies but the combination of AAV and CRISPR has turned out to be quite promising for tackling various problems.