Australian scientists achieve breakthrough in gene-editing therapy that halves ‘Bad’ cholesterol

Daijiworld Media Network - Melbourne

Melbourne, Nov 10: In a world-first human trial, Australian researchers have achieved a major milestone in heart disease prevention with a new gene-editing therapy that dramatically lowers harmful blood fats linked to cardiovascular disease.

The experimental treatment, CTX310, uses CRISPR-Cas9 gene-editing technology delivered through fat-based nanoparticles to switch off the ANGPTL3 gene in the liver — a gene known to regulate levels of LDL (bad) cholesterol and triglycerides.

The Victorian Heart Hospital, operated by Monash Health in collaboration with Monash University, treated three of 15 participants enrolled in the phase 1 global trial, which spans Australia, New Zealand, and the UK, according to a statement from the university on Monday.

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