A team of researchers from the University of California San Diego School of Medicine have developed a new RNA-oriented gene editing technique which could treat hereditary diseases such as ALS and Huntington’s.
The team used a method called RNA-targeting CAS9 (RCas9) to track RNA in live cells and correct “molecular mistakes” which lead to diseases.
The new technique eliminated over 95% of the RNA markers linked to a type of ALS and Huntington’s disease.
Professor of cellular and molecular medicine Gen Yeo said that while this study provides evidence the technique works in a laboratory, there is still a long way to go before it can be tested on patients.
“Before this could be tested in humans, we would need to test it in animal models, determine potential toxicities, and evaluate long-term exposure,” he said.