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

Parkinson's disease causes progressive changes in brain's blood vessels: Study

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New Delhi, (IANS): Researchers in Australia have found that Parkinson's disease causes significant and progressive changes in the brain's blood vessels, changing the understanding of the disease.

While Parkinson's disease is characterised by alpha-synuclein protein deposits, the research demonstrated that region-specific changes to blood vessels in the brain underlie disease progression, Xinhua news agency reported.

"Traditionally, Parkinson's researchers have focused on protein accumulation and neuronal loss, but we have shown the impacts on our cerebrovasculature -- the blood vessels in our brain," said Derya Dik, postdoctoral student at Neuroscience Research Australia (NeuRA).

"Our research identified region-specific changes in the brain's blood vessels, including an increased presence of string vessels, which are non-functional remnants of capillaries," Dik added.

NeuRA researchers, in collaboration with the University of New South Wales and the University of Sydney, also observed changes relating to how blood flows in the brain and how the blood-brain barrier operates.

The findings, published in the journal Brain, may also help open up new treatment avenues.

Researchers believe that targeting these progressive, region-specific changes may be able to slow disease progression and improve outcomes for patients suffering from Parkinson's disease.

In addition to exploring what these findings mean for people with Parkinson's disease, the researchers are considering impacts for other neurodegenerative disorders.

"We are now investigating whether similar cerebrovascular changes are present in post-mortem brain tissue from individuals with Alzheimer's disease and dementia with Lewy bodies tissue," Dik said."This study may lead to new treatment options for people with Parkinson's disease, but we also want to better understand the contribution of vascular pathology in these other neurodegenerative disorders and explore whether this can reveal new targets for therapies and treatments for people with those conditions also," the researcher said. Parkinson's disease causes progressive changes in brain's blood vessels: Study | MorungExpress | morungexpress.com

Read More........→November 28, 2025