Australia leads first human trial of one-time gene editing therapy to halve bad cholesterol

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Melbourne, November 10 (IANS): Researchers in Australia have led a first-in-human trial for a breakthrough gene-editing therapy that halves bad cholesterol and triglycerides in people with difficult-to-treat lipid disorders.

The trial tested CTX310, a one-time CRISPR-Cas9 gene-editing therapy that uses fat-based particles to carry CRISPR editing tools into the liver, switching off the ANGPTL3 gene. Turning off this gene lowers LDL (bad) cholesterol and triglycerides, two blood fats linked to heart disease, according to a statement released Monday by Australia's Monash University.

The Victorian Heart Hospital, operated by Monash Health in partnership with Monash University, treated three of 15 patients aged 18-75 years with difficult-to-treat lipid disorders in phase 1 of the global trial conducted across Australia, New Zealand, and Britain, the statement said, Xinhua news agency reported.

At the highest dose, a single-course treatment with CTX310 resulted in a mean reduction of LDL cholesterol by 50 per cent and triglycerides by 55 per cent, remaining low for at least 60 days after two weeks of treatment, it said, adding LDL cholesterol and triglycerides were reduced by nearly 60 per cent among all participants with various doses, with only mild, short-term side effects reported.

Importantly, CTX310 is the first therapy to achieve large reductions in both LDL cholesterol and triglycerides at the same time, marking a potential breakthrough for people with mixed lipid disorders who have elevations in both, according to the trial published in the New England Journal of Medicine.

"The possibility of a single-course treatment with lasting effects could be a major step in how we prevent heart disease," said Stephen Nicholls, Director of the Victorian Heart Hospital, and study lead investigator."It makes treatment easier, reduces ongoing costs, relieves pressure on the health system, all while improving a person's quality of life," Nicholls said, emphasising plans to focus on larger and more diverse patient populations in future trials of CTX310. Australia leads first human trial of one-time gene editing therapy to halve bad cholesterol | MorungExpress | morungexpress.com

Read More........→December 04, 2025

Scientists Regrow Retina Cells to Tackle Leading Cause of Blindness Using Nanotechnology

Macular degeneration is the leading cause of blindness in developed countries, but regrowing the human cells lost to this condition was the feature of a new successful treatment that took advantage of advances in nanotechnology.

Regrowing the cells of the human retina on a scaffold of synthetic, tissue-like material showed substantial improvements over previously used materials such as cellulose, and the scientists hope they can move on to testing their method in the already blind.

Macular degeneration is increasing in prevalence in the developed world. It’s the leading cause of blindness and is caused by the loss of cells in a key part of the eye called the retina.

Humans have no ability to regrow retinal pigment cells, but scientists have determined how to do it in vitro using pluripotent stem cells. However as the study authors describe, previous examples of this procedure saw scientists growing the cells on flat surfaces rather than one resembling the retinal membrane.

This, they state, limits the effectiveness of transplanted cells.

In a study at the UK’s Nottingham Trent University, biomedical scientist Biola Egbowon and colleagues fabricated 3D scaffolds with polymer nanofibers and coated them with a steroid to reduce inflammation.

The method by which the nanofibers were made was pretty darn cool. The team would squirt polyacrylonitrile and Jeffamine polymers in molten form through an electrical current in a technique known as “electrospinning.” The high voltage caused molecular changes in the polymers that saw them become solid again, resembling a scaffold of tiny fibers that attracted water yet maintained mechanical strength.

After the scaffolding was made, it was treated with an anti-inflammatory steroid.

This unique pairing of materials mixed with the electrospinning created a unique scaffold that kept the retinal pigment cells viable for 150 days outside of any potential human patient, all while showing the phenotype of biomarkers critical for maintaining retinal physiological characteristics.“While this may indicate the potential of such cellularized scaffolds in regenerative medicine, it does not address the question of biocompatibility with human tissue,” Egbowon and colleagues caution in their paper, urging more research to be conducted, specifically regarding the orientation of the cells and whether they can maintain good blood supply. Scientists Regrow Retina Cells to Tackle Leading Cause of Blindness Using Nanotechnology

Read More........→September 26, 2025