Gene editing technology advances toward treating common diseases

Gene therapy is transitioning from a treatment for rare genetic disorders to a potential solution for common conditions such as high cholesterol, heart disease, obesity, and diabetes. While early successes have focused on disorders like sickle cell disease and spinal muscular atrophy, researchers are now exploring genomic therapies that can replace, silence, or edit genes.

In a significant development, scientists at Prime Medicine are conducting a global clinical trial using CRISPR-based prime editing to rewrite DNA directly inside the human body. This trial targets Wilson disease by attempting to edit DNA within liver cells. Unlike traditional CRISPR-Cas9, which cuts both DNA strands, prime editing acts as a ‘search-and-replace’ engine capable of swapping individual DNA letters or inserting new instructions with minimal genomic damage.

Many of these emerging therapies are designed as ‘one-and-done’ treatments, meaning they are intended as single administrations. However, experts note challenges, such as the body’s immune response to viral delivery vehicles, which may prevent subsequent doses if needed.