Researchers at the Cedars-Sinai medical center have devised an experimental agent that “mends” DNA. It also acts as a blueprint for a remedy that restores cell damage resulting from a heart attack, inflammatory, and various other ailments.
Scientists engineered a medication that “fixes” DNA
© Freepik
The study is featured in the journal Science Translational Medicine. Investigators presented the agent TY1—a laboratory iteration of an RNA molecule that stimulates the TREX1 gene. This assists immune cells in “clearing out” damaged segments of DNA.
The treatment was under development for over two decades. Researchers isolated precursor cells from heart tissue. Unlike stem cells, these act more selectively and promote cardiac repair.
Subsequently, the investigators discovered that these precursor cells dispatch microscopic vesicles filled with RNA molecules, termed exosomes. Via these, cells share messages with each other. The objective of the work was to decipher the data inside the exosomes and select molecules possessing therapeutic impact.
Scientists examined the composition of RNA within the exosomes. One RNA molecule significantly outnumbered the others. It turned out that this specific one hastened the recovery of damaged tissues in lab animals following a myocardial infarction.
TY1 is a synthetic rendition of the RNA molecule, fashioned to replicate the structure of RNA-based pharmaceuticals in clinical settings. The preparation functions by boosting the production of immune cells that eradicate damaged DNA, thus minimizing the formation of scar tissue after a heart attack.
TY1 is also potent for other conditions, including autoimmune ones—where the body assaults its own cells. This represents an entirely novel healing pathway. Moving forward, the investigators anticipate progressing to clinical trials.
About the Author
