Britain · UK News Post
A single dose of an experimental jab has been found to cut ‘bad’ cholesterol levels by half within six months of treatment in a small Chinese gene-editing trial, a new study reveals. The therapy is being developed for the treatment of familial hypercholesterolemia, a life-threatening hereditary disease causing abnormally high levels of fats in the blood. Patients with this condition face a heart attack risk 10 to 20 times higher than normal and require lifelong medication.
Researchers at China’s Shanghai Jiao Tong University tested the therapy in six volunteers aged 34 to 62 with confirmed diagnoses. Within six months, patients showed a significant reduction in ‘bad’ cholesterol levels. The experimental treatment also did not cause dangerous side effects, though some participants experienced mild side effects like fever and muscle aches, which resolved within 24 hours.
Medical workers treat an intubated patient (AFP via Getty Images). Low-density lipoprotein cholesterol (LDL-C), also called ‘bad’ cholesterol, is a key driver of heart disease, alongside fat molecules called triglycerides. Currently, most people take daily statin pills to manage these levels.
Scientists have explored gene-editing methods to control fat molecule levels. One approach targets the PCSK9 gene in the liver, which, when overactive, weakens the liver’s ability to remove bad cholesterol. Chinese scientists developed an enzyme from the bacterium *Hafnia paralvei*, modified to target human DNA, specifically the PCSK9 gene in liver cells, to reduce cholesterol production.
Three patients who took the highest dose showed a 74% reduction in PCSK9 enzyme activity and a 52% decrease in ‘bad’ cholesterol levels within six months. Patients are expected to undergo long-term follow-up for up to 15 years. In another trial by the Cleveland Clinic in the US, four volunteers who received the highest dose saw their bad cholesterol drop by 52% and triglycerides fall by 48%.
This therapy targets the ANGPTL3 gene, which regulates fat circulation. Previous research showed that individuals with a mutation inhibiting ANGPTL3 have extremely low cholesterol and triglycerides and rarely develop heart disease. Scientists aim to further develop these gene-editing jabs to treat more patients with various lipid disorders.
Source: The Independent
