Researchers from the United States have introduced a fundamentally novel approach for treating familial hypercholesterolemia. This technique focuses not on removing LDL, or “bad” cholesterol, from the bloodstream, but rather on inhibiting its synthesis within the body. The research was conducted by a team from the Medical University of South Carolina, with their findings detailed in the journal Communications Biology.
The study is centered on a hereditary condition where the liver struggles to eliminate low-density lipoproteins—the so-called “bad” cholesterol. Unlike statins, which enhance the clearance of detrimental lipids from the blood, this new strategy aims to interrupt their production. The scientists target the apolipoprotein B (apoB) protein, as cholesterol particles are incapable of assembling without it.
Experiments were executed using “humanized” mice that had received transplants of human liver cells. This model successfully validated the efficacy of the proposed method. The approach offers a way around the limitations inherent in current medications and holds promise for assisting patients for whom conventional treatments yield inadequate outcomes.
“This represents the essence of personalized medicine: we have discovered a means to engineer a therapeutic agent that functions specifically within the human biological system,” stated lead investigator Steven Duncan.
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