Thorsten Stafforst recalls being discouraged from pursuing RNA editing research a decade ago when CRISPR, a new tool for altering DNA, was gaining popularity. While CRISPR could change DNA, Stafforst and his colleagues at the University of Tübingen in Germany focused on RNA, the close relative of DNA. Critics questioned their interest in RNA editing, but by 2012, they discovered how to edit RNA using enzymes. This work was inspired by creatures like squids, which can naturally rewrite their RNA. Unlike CRISPR, their findings initially received little attention.
Now, RNA editing is growing in the biotech world. Several companies, including big names like Eli Lilly and Roche, are exploring its potential. RNA editing is seen as potentially safer and more adaptable than DNA editing, possibly addressing more diseases, including common ones beyond the reach of current genetic therapies.
However, RNA editing is less tested than CRISPR, and its temporary effects may limit its benefits. Researchers are still figuring out the best ways to use it. RNA molecules play a key role in turning DNA information into proteins. By editing RNA, scientists can alter proteins and potentially treat diseases caused by gene mutations.
Various companies are exploring RNA editing to create medicines. They use different methods to guide enzymes to specific RNA spots to make precise changes. Some companies are even trying to edit larger RNA sections to treat diseases with multiple mutations.
RNA editing offers potential advantages over DNA editing. It allows for reversible changes, making it suitable for chronic conditions or diseases with varying symptoms. It builds on existing RNA-based therapies, aiming to create drugs similar to traditional ones but with the power of gene editing.
Challenges remain, such as ensuring the edits are safe and effective. The field is still in its early stages, with companies testing different approaches. Some have started clinical trials, while others are preparing to do so. As the technology develops, it could eventually lead to new treatments for a range of diseases.