Experimental Gene Editing Treatment Shows Promise in Reducing Bad Cholesterol
In a groundbreaking clinical trial, researchers have achieved a significant reduction in bad cholesterol levels through an experimental gene editing treatment called base editing. This marks the first time the treatment has been used on humans, and initial results are promising.
The study involved ten subjects with congenitally high levels of bad cholesterol. These individuals were administered an injection of VERVE-101, a gene-editing treatment that targets the gene responsible for producing the protein PCSK9. By deactivating this gene, the treatment aims to decrease bad cholesterol levels in the body.
The results of the trial were encouraging. Participants experienced a reduction in bad cholesterol levels by up to 55%. Remarkably, even six months after the treatment, some individuals still maintained lower cholesterol levels. This suggests that the effects of the treatment may be long-lasting.
However, there are certain drawbacks to the gene editing treatment. Some participants reported flu-like symptoms, while others showed a transient surge in liver enzyme levels. Unfortunately, one subject tragically died from a heart attack after receiving the injection. However, a safety panel determined that this unfortunate event was not caused by the gene editing treatment itself.
Despite the setbacks, the biotechnology firm Verve Therapeutics, the creator of VERVE-101, intends to proceed to a phase 2 clinical trial in 2025. The company aims to gather further data to better understand the treatment’s efficacy and safety.
VERVE-101 works by injecting RNA molecules into liver cells, enabling a precise change to occur in the PCSK9 gene. This change deactivates the gene, leading to a reduction in bad cholesterol levels. While the initial results show promise, the long-term impact of gene editing on the human body remains uncertain.
To address this concern, the surviving test subjects will be closely monitored for the next 14 years. This monitoring will provide valuable insights into the potential long-term effects of gene editing on the human body.
The findings of this study could have groundbreaking implications for individuals with high cholesterol levels. However, further research and studies are necessary to fully comprehend and assess the effects of gene editing. The potential benefits of base editing could revolutionize cholesterol management, but ensuring its safety and efficacy is of utmost importance.
