MIT researchers have used specially-developed nanoparticles to turn off specific genes in bone marrow cells, in a development that could lead to a wide range of new treatments.
As bone marrow cells play a pivotal role in producing blood cells, the researchers' nanoparticles could be tailored to help treat heart disease and a number of other ailments, the MIT team explained in a press release.
Nanoparticles targeted at bone marrow
The type of genetic therapy used in the research, called RNA interference, is typically difficult to target towards organs other than the liver, where nanoparticles usually tend to accumulate.
The MIT researchers were able to modify their nanoparticles so that they would accumulate in bone marrow cells rather than in the liver.
"If we can get these particles to hit other organs of interest, there could be a broader range of disease applications to explore, and one that we were really interested in this paper was the bone marrow," Michael Mitchell, a former MIT postdoc and one of the lead authors of the study, explained in the press release.
"The bone marrow is a site for hematopoiesis of blood cells, and these give rise to a whole lineage of cells that contribute to various types of diseases,” he continued.
The researchers tested their hypothesis via a study of mice. In doing so, they showed that they could use their approach to improve recovery for patients after a heart attack by inhibiting the release of bone marrow blood cells. These cells promote inflammation and contribute to heart disease.
'Transformative for disease applications'
The research into nanoparticles has applications in a variety of potential medical treatments. Aside from treating heart disease, the researchers also say it could be used to boost the yield of stem cells in patients who need stem cell transplants.
“If we could develop technologies that could control cellular activity in bone marrow and the hematopoietic stem cell niche, it could be transformative for disease applications,” says Mitchell, who is now an assistant professor of bioengineering at the University of Pennsylvania.
RNA essentially converts the information stored in DNA into proteins. A deeper understanding of the ways this process can be manipulated has the potential to revolutionize the healthcare of the future.