Breakthrough research shows stem cells can be genetically edited within the body

For the first time, scientists have managed to genetically modify stem cells inside the bodies of mice, a feat which could eventually open up new potential in stem cell therapy.

Stem cells can develop into all kinds of other cells, and the body uses these diverse tools for both growth and repair. Because of their remarkable abilities, there's interest in using stem cells for certain medical treatments, but that road is not always easy.

For example, in one particular type of bone marrow transplant, the stem cells that produce blood have to be removed from the body and genetically modified before being transfused back into the patient.

The results of this new study in mice indicate we might be able to remove the risky and complicated extraction step: the necessary genetic edits could be made in vivo, which is likely to be both faster and more effective than the techniques used today.

"When you take stem cells out of the body, you take them out of the very complex environment that nourishes and sustains them, and they kind of go into shock," says the senior researcher behind the study, Amy Wagers from Harvard University in Massachusetts.

"Isolating cells changes them. Transplanting cells changes them. Making genetic changes without having to do that would preserve the regulatory interactions of the cells – that's what we wanted to do."

Ben Reilly wrote:

For the first time, scientists have managed to genetically modify stem cells inside the bodies of mice, a feat which could eventually open up new potential in stem cell therapy.

Stem cells can develop into all kinds of other cells, and the body uses these diverse tools for both growth and repair. Because of their remarkable abilities, there's interest in using stem cells for certain medical treatments, but that road is not always easy.

For example, in one particular type of bone marrow transplant, the stem cells that produce blood have to be removed from the body and genetically modified before being transfused back into the patient.

The results of this new study in mice indicate we might be able to remove the risky and complicated extraction step: the necessary genetic edits could be made in vivo, which is likely to be both faster and more effective than the techniques used today.

"When you take stem cells out of the body, you take them out of the very complex environment that nourishes and sustains them, and they kind of go into shock," says the senior researcher behind the study, Amy Wagers from Harvard University in Massachusetts.

"Isolating cells changes them. Transplanting cells changes them. Making genetic changes without having to do that would preserve the regulatory interactions of the cells – that's what we wanted to do."

https://www.sciencealert.com/breakthrough-research-shows-stem-cells-can-be-genetically-edited-inside-the-body

The article goes on to say that the hope is that techniques like this might someday be used to treat genetic diseases at the source, which would be astounding to say the least.