(Press-News.org) Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders including Duchenne muscular dystrophy, Pompe disease and haemophilia.
Published in the leading journal Cell, the study identifies a previously unknown gateway into human cells, a receptor called AAVR2, that gene therapy viruses use to deliver therapeutic genes. This newly uncovered pathway could allow lower doses of virus to be used in treatment, helping to reduce side effects and treatment costs, while improving patient outcomes.
Gene therapies typically use modified viruses, known as adeno-associated viruses (AAVs), to deliver healthy genes into the body. These treatments have the potential to be life-changing for patients, their families and caregivers. However, they frequently require high vector doses to achieve therapeutic effects which in some cases can trigger severe immune responses, lead to serious complications, or even death.
“We found that certain AAV types can use this newly identified receptor, AAVR2, to enter cells, providing an alternative to the previously known entry route,” said Dr Bijay Dhungel, lead author of the study and researcher at the Centenary Institute’s Centre for Rare Diseases & Gene Therapy and the University of Sydney.
“This discovery uncovers a completely new pathway for delivering genes into cells. Modulating this pathway can potentially make gene therapies safer, cheaper and more precise,” he said.
Using advanced genetic, biochemistry and molecular biology techniques, the researchers showed that AAVR2 plays a crucial role in helping several AAV types, including those widely used in patients, enter cells more efficiently.
“We not only identified this new receptor AAVR2 but also discovered how it binds to the viruses that deliver the genes,” said co-senior author Dr Charles (Chuck) Bailey, Head of the Centre for Rare Diseases & Gene Therapy at the Centenary Institute and researcher at the University of Sydney.
“We then went a step further and engineered a miniature version of the receptor and demonstrated that this significantly enhances how efficiently the gene therapy is taken up in human cells and tissues. We believe this knowledge will ultimately improve the accessibility of gene therapies to patients.”
The researchers say the findings have important implications for the future of gene therapy, offering new strategies to tailor treatments, lower required doses and potentially avoid immune-related complications that have limited some current approaches.
The discovery also advances scientific understanding of how therapeutic viruses interact with human cells. This is essential knowledge for developing the next-generation of safe, effective and precision-guided gene therapies.
The study was supported by funding from NSW Health, National Health and Medical Research Council (NHMRC), Therapeutic Innovation Australia, Tour de Cure, Cure the Future and Brandon Capital CUREator.
[ENDS]
Images:
Dr Chuck (Charles) Bailey and Dr Bijay Dhungel - https://drive.google.com/file/d/1FjkvTn1QvQNOsI8gNpbZj07D5KxvLwPa/view?usp=sharing
Publication:
An alternate receptor for adeno-associated viruses - https://www.cell.com/cell/fulltext/S0092-8674(25)00692-0
For all media and interview enquiries, please contact
Tony Crawshaw, Media and Communications Manager, Centenary Institute on 0402 770 403 or email: t.crawshaw@centenary.org.au
About the Centenary Institute
The Centenary Institute is a world-leading independent medical research institute, closely affiliated to the University of Sydney and the Royal Prince Alfred Hospital. Our research spans the critical areas of cancer, cardiovascular disease, rare diseases, inflammation, infectious diseases, healthy ageing and biomedical AI. Our strength lies in uncovering disease mechanisms and applying this knowledge to improve diagnostics and treatments for patients. For more information about the Centenary Institute, visit centenary.org.au
About the University of Sydney, Australia
As Australia’s first university – founded in 1850 – the University of Sydney has a proud history of global leadership in education and research and inspiring people from all backgrounds to contribute to positive real-world change. We’re a world-renowned teaching and research institution – our research combines the expertise and talents of scholars from many disciplines. Learn more.
END
New route into cells could make gene therapies safer
Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders.
2025-07-15
ELSE PRESS RELEASES FROM THIS DATE:
Team discovers electrochemical method for highly selective single-carbon insertion in aromatic rings
2025-07-15
A research team has discovered an electrochemical method that allows highly selective para-position single-carbon insertion into polysubstituted pyrroles. Their approach has important applications in synthetic organic chemistry, especially in the field of pharmaceuticals.
Their work is published in the Journal of the American Chemical Society on July 14.
“We set out to address the longstanding challenge of achieving single-carbon insertion into aromatic rings with precise positional control,” said Mahito Atobe, Professor, Faculty of Engineering, YOKOHAMA National University. Transformations that modify ...
What cats may teach us about Long COVID
2025-07-15
Feline infectious peritonitis, or FIP, is a serious and historically fatal disease in cats caused by a coronavirus. It behaves in many ways like severe coronavirus infections in humans, causing widespread inflammation, T cell exhaustion, and chronic immune dysfunction. Scientists at the University of California, Davis, have found that mesenchymal stromal cells, or MSC therapy, in combination with antiviral drugs, helped cats’ immune systems recover and reduced systemic inflammation. The study was published in STEM CELLS Translational Medicine.
“Our findings support the idea that FIP in cats is a useful model for studying long-term immune problems after a viral infection — ...
Millions denied life-saving surgery as global targets missed – study
2025-07-14
Progress towards universal access to safe, affordable surgical care is dangerously off track as at least 160 million patients each year are unable to receive surgery - with Low- and Middle-income Countries (LMICs) bearing the brunt of the crisis, a new study reveals.
A global coalition of 60 health experts representing 20 countries is calling for urgent action to resolve the crisis – warning that only 26% of LMICs are on track to meet a target for everyone to be able to access essential surgery within 2 hours, and ...
Record-breaking human imaging project crosses the finish line: 100,000 volunteers provide science with most detailed look inside the body
2025-07-14
There is an accompanying press briefing being hosted by the Science Media Centre in the UK. Please contact UK Biobank for the details.
In a remarkable achievement that is already impacting how we detect and diagnose disease, UK Biobank has completed the world’s largest whole body imaging project, scanning the brains, hearts, abdomens, blood vessels, bones and joints of 100,000 volunteers. These scans, on this scale, show us what is happening in people’s bodies as they age so we can understand how, why and when we get sick.
Since 2015, UK Biobank’s imaging data have been released in batches1 ...
Bio detection dogs successfully detect Parkinson’s disease by odor, study finds
2025-07-14
People with Parkinson’s disease (PD) have an odour that can be reliably detected from skin swabs by trained dogs, a new study has shown.
The research, in collaboration with Medical Detection Dogs and the Universities of Bristol and Manchester, is published in The Journal of Parkinson’s Disease today [15 July].
Two dogs were trained by the charity, Medical Detection Dogs, to distinguish between sebum swabs from people with and without Parkinson’s disease.
In a double blind trial, they showed sensitivity of up to 80% and specificity of up to 98%.
Not only that, ...
Insomnia could be key to lower life satisfaction in adults with ADHD traits, study finds
2025-07-14
UNDER EMBARGO UNTIL 00:01AM UK TIME ON TUESDAY 15 JULY 2025
Insomnia could be key to lower life satisfaction in adults with ADHD traits, study finds
Insomnia could explain why adults with ADHD traits report having a lower quality of life, according to new research led by the University of Southampton and the Netherlands Institute of Neuroscience.
A study published today [15 July] in BMJ Mental Health found that having higher ADHD traits was associated with a lower quality of life and that insomnia could be part of the reason why.
At least ...
Study discusses how to mitigate damage from gunshot injuries to the brain in children and young adults
2025-07-14
FOR IMMEDIATE RELEASE: July 14, 2025
CONTACT: Camille Jewell
cjewell@vancomm.com or 202-248-5460
NASHVILLE — A study presented today at the Society of NeuroInterventional Surgery’s (SNIS) 22nd Annual Meeting found that almost half of gunshot wound injuries to the brain in children and young adults include penetrating cerebrovascular injuries (PCVIs). These types of injuries, which damage blood vessels in the brain and may result in high rates of permanent disability and death, may be mitigated if hospitals ...
New research challenges animal dietary classifications in Yellowstone National Park
2025-07-14
PROVIDENCE, R.I. [Brown University] — Scholars and schoolchildren alike have generally classified animals by the foods they eat: carnivores eat meat; browsers consume flowering plants, conifers and shrubs; and grazers focus on grasses.
But a new federally funded study led by Brown University biologists and scientists at Yellowstone National Park revealed that different circumstances lead herbivores to eat a much wider variety of plants than previously believed.
Published in the Proceedings of the National Academy of Sciences, the new research suggests that the traditional classification schemes that distinguish herbivores by their ...
Parenthood not lessening loss for widowed people, 25 years of interviews suggest
2025-07-14
Widowed parents who enjoy close relationships with their adult children still struggle with loneliness, according to the first study of its kind.
Published in the peer-reviewed journal Aging & Mental Health, the analysis spanning 25 years was based on interviews with more than 5,500 men and women including those whose spouse had died.
The findings contradict commonly held assumptions that indicate widowed parents experience much lower levels of loneliness than those without children.
Although the bond between bereaved women and their offspring is strengthened, the authors of this new study say this is insufficient to fill the emotional void left after the death of ...
UC Irvine astronomers discover scores of exoplanets may be larger than realized
2025-07-14
Irvine, Calif., July 14, 2025 — In new research, University of California, Irvine astronomers describe how more than 200 known exoplanets are likely much larger than previously thought. It’s a finding that could change which distant worlds researchers consider potential harbors for extraterrestrial life.
“We found that hundreds of exoplanets are larger than they appear, and that shifts our understanding of exoplanets on a large scale,” said Te Han, a doctoral student at UC Irvine and lead author of the new Astrophysical Journal Letters study. “This means we may have actually found fewer Earth-like planets so far than we thought.”
Astronomers ...
LAST 30 PRESS RELEASES:
Making regular GPS ultra-precise
Webb Telescope unveils doomed star hidden in dust
UT Southwestern preventive cardiologist to receive the 2025 Chairman’s Award
Slime mold metabolites are a promising, eco-friendly repellent of root-knot nematodes
Pathological mechanism of mechanosensitive cells driving the growth of keloids
First large-scale Alzheimer disease study in brain tissue from African American donors implicates roles for many novel genes
In a nasal spray, gold “nanoparticles” deliver a targeted treatment to the brain. A potentially revolutionary approach to mental disorders and neurodegenerative diseases already has a patent
Current and recommended diets in the USA have embedded forced labor risk
AI breakthrough helps astronomers spot cosmic events with just a handful of examples
New vaccine shows promise against typhoid and invasive salmonella in first human trial
Engineered “natural killer” cells could help fight cancer
New 3D printing method ‘grows’ ultra-strong materials
Lizard genetics provide new perspective on evolution
Can a Stevia-derived sweetener improve hair loss treatment?
Method to assess the status of wild reindeer may help with conservation efforts
Do imported cut flowers spread livestock viruses?
Does prior incarceration contribute to poor health later in life?
Could slime mold microbes be a source of potent antimicrobials?
Record-breaking 2024 Amazon fires drive unprecedented carbon emissions and ecosystem degradation
Birds thrive despite pollution from ‘forever’ chemicals
Deadwood brings wild orchids to life
Changes in gut microbiota influence which patients get AIG-related neuroendocrine tumors
Medicaid expansion linked to improved long-term survival in cancer patients
Women with surgical menopause may exit workforce earlier, but hormone therapy could help
Trailblazing Young Scientists honored with $250,000 prizes at Blavatnik National Awards Gala
Revolutionary blood test for ME / Chronic Fatigue unveiled
Calorie labelling linked to 2% average reduction in energy content of menu items
Widely prescribed opioid painkiller tramadol not that effective for easing chronic pain
Exercise snacks may boost cardiorespiratory fitness of physically inactive adults
15,000 women a year with breast cancer could benefit from whole genome sequencing, say researchers
[Press-News.org] New route into cells could make gene therapies saferScientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders.