A safer way to treat genetic diseases

Marc Güell

Universitat Pompeu Fabra (UPF), Barcelona, Spain

Therapeutic applications of human genome engineering are widening —from correcting errors in the genome that cause genetic diseases to delivering therapeutic payloads, such as CAR-T immunotherapies for cancer and autoimmune conditions. Until now, most solutions have relied on DNA-based methods, which can sometimes cause unwanted toxicities and have administration difficulties.

Recently, a new approach has been developed that uses RNA instead of DNA to help address these challenges. RNA is a natural messenger in our cells that carries instructions for building and repairing the body. By using RNA, the team has found a gentler and more precise way to add healthy information that it is needed, reducing the risk that the body will react badly.

This new technology is inspired by how nature works. Researchers looked at different animals and discovered a tool in a rare monkey that could do this job much better than previous methods. With this natural discovery, they created a new way to help cells fix the problems caused by errors in their DNA. It is especially helpful for diseases that do not have good treatments right now.

The main goal of this discovery is to provide a next generation tool for safely writing in genomes. This new method avoids complicated or risky procedures and prevents large, hard-to-control changes in the body.

In summary, this new RNA-based technology makes it possible to write therapeutic messages in genomes in a safer, easier, and more reliable way. It brings us closer to a future where genetic medicines can be deployed successfully, giving new hope to patients and their families.

LINE-1 Identification for novel elements with active retrotransposition

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