Experimental Gene Editing Treatment Shows Promise in Reducing Bad Cholesterol Levels
In a groundbreaking breakthrough, researchers have successfully utilized an experimental gene editing treatment to significantly decrease levels of bad cholesterol in human subjects. The technique, known as base editing, was carried out on humans for the very first time, signaling a major advancement in the field of gene therapy.
The treatment involved administering VERVE-101, a gene editing treatment, to the subjects. VERVE-101 works by deactivating the gene responsible for regulating LDL cholesterol levels, commonly referred to as bad cholesterol. Remarkably, test subjects witnessed a substantial reduction in their LDL cholesterol levels, with some experiencing a decrease of up to 55% after just 28 days.
Encouragingly, individuals who received a high dosage of VERVE-101 continued to have lower LDL cholesterol levels even six months after the initial injection. This suggests that the treatment could have a lasting impact on cholesterol regulation.
While the results are promising, the treatment did come with a few drawbacks. Participants reported experiencing flu-like symptoms and temporary elevations in liver enzyme levels. However, a safety panel promptly addressed concerns, affirming that a fatal heart attack experienced by one participant was not caused by VERVE-101.
Looking ahead, Verve Therapeutics, the company behind this groundbreaking research, plans to conduct a phase 2 clinical trial of VERVE-101 in 2025. This trial aims to further explore the safety and efficacy of the treatment in a larger group of participants.
The treatment involves the use of two RNA molecules encapsulated in a lipid nanoparticle. These molecules specifically target and edit the PCSK9 gene, found in liver cells. By altering this gene, the treatment successfully controls cholesterol production, leading to a reduction in LDL cholesterol levels.
However, it is vital to note that the long-term effects of gene editing on the human body are still largely unknown. Therefore, the research team intends to closely monitor and follow the test subjects for the next 14 years to gain valuable insights into the treatment’s long-term impact.
The successful utilization of base editing as a gene therapy tool presents a significant breakthrough in the battle against high cholesterol levels. With further research and clinical trials, this gene editing technique could potentially revolutionize the treatment of cardiovascular diseases and bring hope to millions worldwide suffering from high levels of bad cholesterol.
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