New CRISPR Gene-Editing Therapy for Permanent Cholesterol Reduction

 



A breakthrough CRISPR-based gene-editing therapy has shown encouraging results in reducing cholesterol permanently by targeting the ANGPTL3 gene. Early findings from a small clinical trial suggest that this treatment could provide a long-term solution for patients struggling with severe or treatment-resistant high cholesterol.

Targeting the ANGPTL3 Gene

The experimental therapy works by disabling the ANGPTL3 gene, which regulates levels of low-density lipoprotein (LDL) and triglycerides in the bloodstream. Known as “bad cholesterol,” LDL contributes to plaque buildup in arteries, while triglycerides are associated with an increased risk of heart disease. In the initial study involving 15 patients, those given the highest dose experienced a 50% reduction in LDL cholesterol and a 55% drop in triglycerides.

Inspired by a Natural Mutation

The scientific concept behind the treatment arises from a rare natural mutation affecting about one in every 250 people. Individuals with a non-functional ANGPTL3 gene naturally maintain low cholesterol and triglyceride levels without adverse effects. The therapy aims to mimic this condition by editing liver cells, the body’s primary site of cholesterol regulation, while minimising the risk of affecting other tissues.

Safety and Early Findings

The first human trial primarily assessed the treatment’s safety. Most participants reported only mild, temporary side effects, such as infusion-related reactions. One participant with advanced cardiovascular disease died months after receiving a low dose, though investigators confirmed it was unrelated to the therapy. Regulatory authorities have approved further trials to study long-term safety and effectiveness, with participants monitored for up to 15 years.

If proven effective, this one-time gene-editing therapy could transform cardiovascular disease prevention. It may offer an alternative to lifelong medication, especially for individuals who cannot tolerate statins or other cholesterol-lowering drugs. However, experts caution that further large-scale studies are necessary before the therapy becomes a mainstream treatment option.

#CRISPR
#GeneEditing
#CholesterolControl
#GeneticTherapy
#PrecisionMedicine
#HeartHealth
#Biotechnology
#MedicalInnovation
#GenomicMedicine
#FutureOfMedicine


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