According to the researchers, the development could lead to a new generation of drugs to precisely treat a range of diseases.
"In the future we hope to apply this strategy to target other disease-causing RNAs, which range from incurable cancers to important viral pathogens such as Zika and Ebola," said lead author Sai Pradeep Velagapudi, research associate at The Scripps Research Institute (TSRI) in the US.
The findings demonstrated that the compound, known as Targaprimir-96, triggers breast cancer cells to kill themselves via programmed cell death by precisely targeting a specific RNA that ignites the cancer.
"The study represents a clear breakthrough in precision medicine, as this molecule only kills the cancer cells that express the cancer-causing gene -- not healthy cells. These studies may transform the way the lead drugs are identified -- by using the genetic makeup of a disease," Matthew Disney, professor at TSRI, added.
Since the compound was highly selective in its targeting, healthy cells were unaffected.
In contrast, a typical cancer therapeutic drug targets and kills cells indiscriminately, often leading to side effects that can make these drugs difficult for patients to tolerate, Disney noted.
"This is the first example of taking a genetic sequence and designing a drug candidate that works effectively in an animal model against triple negative breast cancer," Matthew Disney, professor at TSRI, added.
The study was published online in the journal Proceedings of the National Academy of Sciences.
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