Published on November 17, 2023, 5:20 am

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Researchers have made a significant breakthrough in treating high levels of bad cholesterol in humans using an experimental gene editing treatment called base editing. This groundbreaking technique has been performed on humans for the first time and has shown promising results.

In a clinical trial, 10 individuals with congenitally high levels of low-density lipoprotein (LDL), commonly known as bad cholesterol, were injected with VERVE-101, a gene-editing treatment that utilizes the base editing technique. This treatment targeted the gene responsible for producing the PCSK9 protein found in the liver, which regulates LDL.

The results were remarkable. After just 28 days, test subjects saw their LDL levels reduced by as much as 55 percent. Before the experiment, their average LDL level was dangerously high at 193 mg/dL. According to medical standards, people should ideally have less than 100 LDL.

Even six months after receiving the injection, participants who had received a higher dose of VERVE-101 continued to benefit from lower LDL levels. University of Pittsburgh cardiologist Ritu Thamman noted that this level of improvement is rarely seen with statin drugs, which are commonly used to manage cholesterol levels.

However, it is important to consider potential drawbacks associated with this novel treatment. Participants in the trial experienced temporary flu-like symptoms such as chills, fever, and headaches. Additionally, there was a transient surge in liver enzyme levels. Unfortunately, one participant passed away due to a heart attack approximately five weeks after receiving VERVE-101. Another participant also suffered a non-fatal heart attack shortly after the injection. However, Nature reported that an independent safety panel determined that these incidents were not directly related to VERVE-101 but rather pre-existing advanced heart disease.

Verve Therapeutics, a biotechnology firm based in Boston, Massachusetts plans to initiate a phase 2 clinical trial of VERVE-101 in 2025. This next phase aims to further evaluate the safety and effectiveness of the treatment.

VERVE-101 consists of two molecules of RNA enclosed in a lipid nanoparticle. One molecule identifies the PCSK9 gene while the other molecule edits the gene itself. When VERVE-101 is injected into a human subject, it targets liver cells, where the RNA editing molecule precisely alters one of the nucleotide bases of the PCSK9 gene. This prompts the deactivation of the PCSK9 gene, leading to a reduction in LDL levels.

While this research milestone is exciting, there are still many unknowns regarding how gene editing will impact other aspects of the human body. The research team plans to observe the surviving test subjects for up to 14 years to gain further insights.

Overall, this innovative base editing technique shows great promise in treating high cholesterol levels by directly targeting and altering genes responsible for its regulation. As research continues, it has the potential to revolutionize how we approach various genetic conditions and pave the way for more personalized treatments.


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