According to the National Institute of Health, gene therapy is an experimental technique that uses genes to treat or prevent disease. This technique includes but is not limited to inserting a gene into another’s cells, inactivating a mutated gene or replacing a mutated gene. At King’s College London, researchers used microRNA-199a to invigorate cardiac repair in pigs. The treated animals demonstrated noticeable improvements such as increased muscle mass and reduced scarring.
MicroRNAs are small non-coding RNA molecules involved in the regulation of gene expression. To do so, they bind to a target mRNA in order to prevent protein production. It is speculated that microRNAs regulate about 30% of human protein coding genes. MicroRNAs also aid in the regulation and reprogramming of stem cells.
PHOTOGRAPH BY SIMONE VAN DEN BERG, DREAMSTIME
However, many pigs did not benefit from the gene therapy. Persistence and uncontrolled amounts of the gene therapy led to a sudden arrhythmic death of the pigs. Although the numbers favor the undesired side of this breakthrough, researchers are hopeful for a better outcome next time with strict control regarding the dosages for this therapy.
In my opinion, this study opens the door for more research regarding the heart. It shows how effective microRNAs can be when used properly. As lead author, Mauro Giacca, said, “It is a very exciting moment for the field. After so many unsuccessful attempts at regenerating the heart using stem cells, which all have failed so far, for the first time we see real cardiac repair in a large animal.” This is mostly exciting because of how closely related a pig’s heart is to a human heart. Does that mean humans are next?