CRISPR Developments Highlight Potential Unintended Consequences in Genetic Editing

The United States Food and Drug Administration has approved the first-ever clinical trial using CRISPR-Cas13 RNA editing, targeting wet age-related macular degeneration, a condition affecting millions of older individuals worldwide.

This trial represents a significant milestone in gene therapy, as it focuses on RNA rather than DNA, which could mitigate some risks associated with genetic editing. RNA editing, unlike DNA editing, offers a transient approach, altering how RNA interprets genetic instructions without permanently changing a person's DNA, thus potentially reducing unintended consequences.

Wet age-related macular degeneration, which affects over two hundred million people globally, is often treated with regular injections that block vascular endothelial growth factor to control abnormal blood vessel growth.

The new RNA editing therapy aims to deliver a one-off injection that could significantly reduce the need for monthly treatments, marking a promising advance in medical therapies. Researchers, including Flora Hui at the Centre for Eye Research Australia and Guei-Sheung Liu, are optimistic about the implications of RNA editing, emphasizing its potential to safely develop new therapies for various conditions.

However, as with all gene editing technologies, ongoing research is essential to address potential risks and ensure the safety of these innovative treatments. This trial not only sets the stage for future advancements in RNA editing but also highlights the ongoing dialogue regarding the benefits and challenges of genetic modifications in medicine.

According to the Genetic Literacy Project, the technology is still in its early stages, but the approval of this trial signifies the arrival of RNA editing therapies in clinical settings.