A genetic technique called base editing is used to modify the PCSK9 gene in humans, resulting in lower levels of low-density lipoprotein cholesterol in the bloodstream.

We analyzed the available evidence on base editing of the PCSK9 gene and its effect on LDL cholesterol in humans. What we’ve found so far is that one assertion supports the idea that using base editing to modify the PCSK9 gene leads to lower levels of LDL cholesterol in the bloodstream [1]. No assertions in our review contradicted this claim. Base editing is a precise form of gene editing that changes a single letter in the DNA code without cutting the entire strand. The PCSK9 gene plays a role in how the body clears LDL cholesterol — often called “bad” cholesterol — from the blood. When this gene is altered, it can reduce the amount of LDL circulating in the bloodstream. The single assertion we reviewed described this effect as observed in human participants. Because we only reviewed one assertion, and no studies or data points were provided beyond that, we cannot say how consistent or widespread this effect is across different people or settings. We also cannot determine how long the effect lasts, whether it’s safe over time, or if it works the same in all populations. The evidence we’ve reviewed leans toward the possibility that base editing the PCSK9 gene can lower LDL cholesterol in humans, but the amount of data is very limited. More research would be needed to understand the full picture. For now, if you’re considering new treatments for high cholesterol, this approach is still experimental and not available outside of clinical trials. Always talk with your doctor about proven, evidence-based options for managing cholesterol levels.