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ARCHIVE
* 2024 GERARD CROCK LECTURE: PROFESSOR JEAN BENNETT
Video
2024 GERARD CROCK LECTURE: PROFESSOR JEAN BENNETT
WATCH PROFESSOR JEAN BENNETT PRESENT THE 15TH GERARD CROCK LECTURE ON
PIONEERING TREATMENTS FOR BLINDING RETINAL DISORDERS.
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Watch pioneering ocular gene therapy researcher Professor Jean Bennett
present an enlightening presentation about the sight-saving potential of gene
therapy at the 15th annual Gerard Crock Lecture.
The Gerard Crock Lecture series honours the memory of renowned
ophthalmologist, the late Professor Gerard Crock, inaugural Ringland Anderson
Professor of Ophthalmology at the University of Melbourne.
Professor Bennett is an international authority on the development of gene
therapies to treat inherited retinal diseases, which are the most common
cause of blindness in working-age people.
She has received numerous international awards including the prestigious
Champalimaud Vision Award.
Professor Bennett shares her story leading pivotal research that led to the
development of the world’s first approved ocular gene therapy – Luxturna, a
treatment for a rare genetic form of childhood blindness Leber Congenital
Amaurosis.
MEET OUR PRESENTER
--------------------------------------------------------------------------------
About
PROFESSOR JEAN BENNETT
Professor Bennett is an international authority on the development of gene
therapies to treat inherited retinal diseases, which are the most common
cause of blindness in working-age people.
Read More
* WATCH THE COMMUNITY FORUM AND MINI VISION EXPO
Video
WATCH THE COMMUNITY FORUM AND MINI VISION EXPO
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On October 4, our community came together to celebrate the launch of our
Consumer Advisory Group and engage directly with the science that’s shaping
tomorrow’s treatments.
Watch the recording to discover how your involvement can influence the future
of eye research. You’ll also hear from our researchers about the latest
advancements in vision science and meet our friends from Vision Australia and
Guide Dogs Victoria.
Hear from experts:
* * CERA Board Director and Chair of the CERA Consumer Advisory Group Simon
Brewin
* Cerulea Clinical Trials Chief Executive Officer Michelle Gallaher
* Retinal Gene Therapy Unit Principal Research Fellow Associate Professor
Lauren Ayton
Hosted by Glaucoma Research Fellow Dr Flora Hui.
MEET OUR PRESENTERS
--------------------------------------------------------------------------------
People
SIMON BREWIN
Simon Brewin is an experienced non-executive director with expertise in
governance, infrastructure and project management. He was appointed to the
Royal Victorian Eye and Ear Hospital board in 2017 and is Deputy Chair of the
Audit and Risk Committee and member of the Quality & Safety Committee.
Read More
People
MICHELLE GALLAHER
Michelle Gallaher is an award-winning thought leader, speaker, advocate and
health technology entrepreneur. She has established four startups, including
Trialkey which applies artificial intelligence to improving and predicting
clinical trial results.
Read More
People
ASSOCIATE PROFESSOR LAUREN AYTON
Associate Professor Lauren Ayton is a member of CERA’s Executive team and
co-leads the Retinal Gene Therapy Unit and VENTURE inherited retinal diseases
(IRD) registry with Dr Tom Edwards. She is also the Head of the Vision
Optimisation Unit at the University of Melbourne.
Read More
People
DR FLORA HUI
Dr Flora Hui is a clinician scientist with a research interest innovative
methods to improve patient outcomes in optometry and ophthalmology and
neuroprotectants for treating glaucoma.
Read More
* NEW RESEARCH FUNDING TO IMPROVE OUTCOMES FOR WOMEN
News
NEW RESEARCH FUNDING TO IMPROVE OUTCOMES FOR WOMEN
CERA’S WORLD-LEADING RESEARCH INTO FEMALE CARRIERS OF X-LINKED INHERITED
RETINAL DISEASES HAS BEEN GIVEN A VITAL FUNDING BOOST THANKS TO THE FELTON
BEQUEST.
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CERA has received $300,000 over three years from The Felton Bequest to
advance our groundbreaking research on female carriers of X-linked inherited
retinal diseases (IRDs).
With this funding, Dr Sena Gocuk will lead two new research projects with the
support of CERA Principal Investigators Associate Professor Lauren Ayton and
Dr Thomas Edwards.
The first study aims to establish a clinical test to identify a carrier’s
risk of severe vision loss, and the other will investigate the safety and
efficacy of gene therapy in these women.
“Treatment at this stage is focused on males with X-linked conditions. What
we’re trying to find out is whether the treatment available for men in
current clinical trials will work on women as well,” says Dr Gocuk.
“I’m so honoured that we’ve received this support from The Felton Bequest. It
allows us to explore the questions that remain unanswered for women living
with these eye diseases.”
UNDERSTANDING FEMALE CARRIERS OF X-LINKED IRDS
IRDs are a broad group of eye conditions that result from a change or
‘mutation’ in one or more genes. X-linked IRDs, such as choroideremia and
X-linked retinitis pigmentosa, are caused by a gene mutation on the X
chromosome.
Males, who have both an X and a Y chromosome, will develop the disease if
their X chromosome carries the mutated gene. This typically results in severe
vision impairment.
Female carriers of X-linked IRDs also have the mutated gene on their X
chromosome. But because they have two X chromosomes – one mutated and one
healthy – the disease affects them differently.
“The term ‘carrier’ implies they only carry the mutated gene to pass it on to
their son. But we now know that carriers can experience vision loss, and some
are even as severely affected as males,” says Dr Gocuk.
Dr Gocuk’s previous research has shown that disease severity in female
carriers depends on how much of each X chromosome is being expressed.
If most of the cells in the retina are expressing the mutated gene, they will
have severe retinal disease. If cells are mostly expressing the normal gene,
then they’re relatively unaffected.
Dr Gocuk and her team are now building on this knowledge to explore how
female carriers can be better diagnosed and whether they are suitable
candidates for emerging treatment options.
Better access: Dr Sena Gocuk ultimately wants to ensure female carriers of
X-linked IRDs can access potential treatments.
GETTING FEMALE CARRIERS INTO CLINICAL TRIALS
Currently, there are no approved treatments for X-linked IRDs. However, gene
therapy – which replaces a defective gene with a healthy copy – is a
promising option.
While there are clinical trials for X-linked IRDs currently underway, women
are usually excluded because there is no safety data on the effect of gene
therapy in female carriers.
“Males with X-linked conditions don’t have a functional gene so gene therapy
just replaces the gene that’s missing. But in women, we need to understand
how an introduced gene will interact with the existing ones,” says Dr Gocuk.
To investigate this, Dr Gocuk and her team are collaborating with Head of
Genetic Engineering Research Associate Professor Guei-Sheung (Rick) Liu and
Dr Livia dos Santos Carvalho from the University of Melbourne – experts in
preclinical models for IRDs.
Initial studies will explore the effects of gene therapy using cells that
mimic the genetic profile of carriers. Once they understand what’s happening
at a cellular level, they will use mouse models to assess whether gene
therapy can slow disease progression.
“We’re hoping this lab work will give us a clearer idea of whether there are
any implications or toxic effects of having that gene introduced, and
therefore, whether it is feasible for female carriers to be included in
future gene therapy trials,” says Dr Gocuk.
GREATER CERTAINTY FOR FEMALE CARRIERS
The research also aims to develop a saliva or cheek swab test for female
carriers that shows how the genes are being expressed between the two X
chromosomes. This will offer insights into their disease severity and how it
is likely to progress over time.
Through a collaboration with Dr Quentin Gouil and Professor Marnie Blewitt at
the Walter Eliza Hall Institute (WEHI), the team is using readily available
peripheral DNA in saliva, cheek swabs and blood to determine which sample
gives the best indication of what’s happening in the retina.
“I’m hoping this data will help us develop a very simple lab test to guide
carriers on what they can expect – whether they will remain on a mild
trajectory of retinal disease or progress to severe retinal degeneration,”
says Dr Gocuk.
“This information would be very valuable for the many carriers we know feel
anxious about their own vision.”
ADVOCATING FOR EQUALITY
Dr Gocuk’s research is motivated by her desire to ensure female carriers have
access to the same support and treatment opportunities as men with X-linked
IRDs.
“Once I understood the issues carriers were facing, like being dismissed,
misdiagnosed or diagnosed too late, I saw there was a need to advocate for
these women.”
“I became very passionate about raising awareness that carriers can be
affected, as well as helping clinicians to better support them,” she says.
“There’s definitely a growing interest in female carriers, but we need the
time and resources to do these research projects. Funding from The Felton
Bequest helps us achieve that, and we sincerely thank them for the support.”
RELATED PAGES
--------------------------------------------------------------------------------
Research
UNDERSTANDING INHERITED RETINAL DISEASES
New research is revealing more about female carriers of inherited retinal
disease. And its findings are challenging the long-held idea they don’t
experience vision symptoms as severely as males do.
Read More
Research
WOMEN AT RISK OF PASSING VISION LOSS TO THEIR CHILDREN LACK SUPPORT
A global survey of female carriers of genetic eye diseases has shown a lack
of counselling and support for women at risk of passing the conditions onto
their children.
Read More
* A RESEARCH SCHOLARSHIP SAYS THANK YOU FOR A LIFETIME OF CARE
Stories
A RESEARCH SCHOLARSHIP SAYS THANK YOU FOR A LIFETIME OF CARE
MELBOURNE WOMAN SHELLEY KLINE HAS RAISED MORE THAN $500,000 FOR RESEARCH INTO
EYE DISEASE AS A TRIBUTE TO THE ICONIC PROFESSOR FRANK BILLSON. CERA
RESEARCHERS ARE AMONG THOSE BENEFITTING FROM THE GENEROUS SCHOLARSHIP.
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Shelley Kline has spent four decades dealing with pain, medication, and
traumatic procedures for her eye disease. She knows research is the key to
better treatment and care for children and young people today.
Shelley, now 52, was diagnosed at the age of eight with uveitis, a form of
eye inflammation that affects the middle layer of tissue in the eye wall.
Uveitis is the term used to describe a broad number of inflammatory diseases
that produce eye swelling and redness and destroy eye tissue.
In Shelley’s case, the disease led to extreme pain and, eventually,
enucleation (removal of the eye globe) of her right eye in 2018.
“As a child, I remember my mother crying when my Melbourne doctor told us
about my condition and what might lie ahead,” Shelley said.
Soon after the diagnosis, Shelley and her parents met Professor Frank
Billson, or ‘Prof’ as she refers to him, one of the only specialists in
Australia with expertise on uveitis.
Prof Billson AO, who is now in his nineties, had a distinguished career in
ophthalmology, which began in Melbourne. He was the Director of the
Ophthalmology Department for seven years at the Royal Children’s Hospital
(RCH) before moving to Sydney.
Shelley and her family regularly travelled to the Sydney Eye and Ear Hospital
where Prof Billson was working, for treatment.
“My parents and I just fell in love with him because he had such a beautiful
bedside manner,” Shelley says. “He always asked how I was going at school, if
I had good friends, and if I enjoyed sport – he was interested in all
elements of my life and family.”
Shelley was eight when she first met Professor Billson.
Throughout her life, Shelley has needed frequent visits to ophthalmologists,
and in her school years she would visit a doctor weekly to manage the
inflammation and check for signs of glaucoma.
After leaving school, Shelley completed Arts and Social Work degrees before
living in Israel for several years. She returned to Australia when her
condition worsened and she needed the support of her parents, who dedicated
themselves to her treatment and care.
Back in Melbourne, Shelley married and had three children and underwent
numerous procedures, as well as treatment with steroids and medication for
her uveitis and related conditions glaucoma, corneal edema, and juvenile
rheumatoid arthritis.
In 2018, no more could be done for her right eye. With the guidance of Prof
Billson, who she consulted on major treatment decisions, Shelley made the
difficult call to have the eye removed.
“The eye wasn’t functioning properly, it was almost blind, and I could no
longer manage the pain,” said Shelley. “Throughout my whole life, whenever
there have been complicated decisions to make about my eye, Prof always took
the time to research, empathise, be compassionate, and supportive.
“I still wake up in pain every day, but research is key. If we knew now then
what we know now, things would have been different for me.”
Professor Billson’s care and kindness over more than four decades was so
significant that Shelley has now established the Professor Frank Billson
Research Scholarship. It provides funding for a staff member within the
Ophthalmology department at the RCH. Dr Sandra Staffieri AO, who is a
Research Fellow at CERA and the Retinoblastoma Care Co-ordinator/Senior
Clinical Orthoptist at the hospital, was one of the inaugural recipients.
“Securing research funding is highly competitive and even more so when the
disease is rare and the impact of the outcomes of the research will affect so
few people,” she said. “It was validating to be reassured that even patients
with a rare condition were important, and I am so proud to have been able to
be part of this work.”
CERA Managing Director Professor Keith Martin said Shelley’s establishment of
the research scholarship was a significant contribution to building knowledge
in the field.
“Professor Billson is an extraordinary person who continues to make a
different to countless children and their families over many years,” he said.
“CERA is grateful that our researchers can also benefit from the
scholarship.”
Shelley is excited about future research into children’s eye disease.
“I love meeting and talking to people and telling them about Professor
Billson and the impact he has had on my life, and many others,” she said.
“It was so wonderful to take my husband Adam Joel and my three beautiful
children to meet Prof a few years ago to show him that, despite everything,
look what I have created.
“This scholarship honours Prof’s legacy to medicine forever and will help
children for generations to come.”
RELATED PAGES
--------------------------------------------------------------------------------
Science and Research
UVEITIS AND RETINAL VASCULAR DISEASE RESEARCH
Our researchers are devoted to investigating new treatments for this painful
and blinding inflammatory eye condition, as well as advancing our scientific
understanding of its causes and prognosis.
Read More
Support our work
FUNDRAISE FOR CERA
Here are a few ways you can help support our world-leading research through
fundraising.
Read More
* NEW RESEARCH SHOWS POTENTIAL OF GENE THERAPY TO REPLACE REGULAR EYE
INJECTIONS
Research
RESEARCH SHOWS GENE THERAPY’S POTENTIAL TO REPLACE EYE INJECTIONS
AUSTRALIAN SCIENTISTS HAVE SUCCESSFULLY USED AN INNOVATIVE GENE THERAPY
TECHNIQUE IN THE LAB TO COMBAT A KEY CAUSE OF VISION LOSS IN ‘WET’
AGE-RELATED MACULAR DEGENERATION AND DIABETIC EYE DISEASE.
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Their research, published in the Proceedings of the National Academy of
Sciences, highlights the potential of gene therapy to provide a new
alternative treatment to regular eye injections.
The pre-clinical research was led by Satheesh Kumar and Associate Professor
Guei-Sheung (Rick) Liu from the Centre for Eye Research Australia (CERA) and
University of Melbourne, along with scientists from the University of Sydney,
Children’s Medical Research Institute, University of Western Australia and
Zhongshan City People’s Hospital, China.
The researchers for the first time used an RNA editing tool – known as CRISPR
Cas13 – to suppress the production of Vascular Endothelial Growth Factor
(VEGF) in human retinal cells.
VEGF is a protein that causes abnormal leaky blood vessels to grow in the
retina at the back of the eye – and is the key driver of vision loss in
diseases such as ‘wet’ age-related macular degeneration and diabetic
retinopathy that affect 200 million people worldwide.
Associate Professor Liu said the new research demonstrated the potential to
develop a gene therapy using CRISPR Cas13 to control VEGF in the retina.
“Our study shows the potential of RNA editing to develop gene therapies that
offer an alternative treatment to the invasive, frequent eye injections that
are currently used to treat wet macular degeneration and diabetic eye
disease,’’ he said.
Associate Professor Guei-Sheung (Rick) Liu is developing an alternative to
regular eye injections.
The experiment targeted the mRNA sequence that instructs cells to produce
VEGF. It delivered the RNA editing tool via an AAV viral vector and was
tested on a mouse model and human retinal cells derived from stem cells.
It showed that the viral vector was effective in delivering the treatment to
retinal cells and produced a significant reduction in VEGF, and a slowing of
disease progression in the mouse model.
Satheesh Kumar said this research informs future work to develop a more
tailored approach to delivering gene therapies to patients.
“RNA editing enables us to change the genetic instructions that influence the
way cells behave without permanently altering their DNA,’’ he said. “This
could allow treatments to be adjusted over time, depending on clinical need.”
Associate Professor Liu said the research aims to make a difference for
people with eye disease who face the prospect of having eye injections every
six to 12 weeks for the rest of their lives.
“Although this research is in the early discovery stages and requires further
development before transitioning to clinical trials, we envision that RNA
editing could become a viable alternative to invasive and costly eye
injections that have become a fact of life for many people living with wet
macular degeneration or diabetic eye disease.’’
READ THE STUDY
Satheesh Kumar, Yi-Wen Hsiao, Vickie H Y Wong, Deborah Aubin, Jiang-Hui Wang,
Leszek Lisowski, Elizabeth P Rakoczy, Fan Li, Luis Alarcon-Martinez, Anai
Gonzalez-Cordero, Bang V Bui, Guei-Sheung Liu. Characterisation of RNA
editing and gene therapy with a compact CRISPR-Cas13 in the retina,
Proceedings of the National Academy of Science doi/10.1073/pnas.2408345121
MEDIA CONTACT
Janine Sim-Jones, Centre for Eye Research Australia, jsimjones@cera.org.au,
0420 886 511
RELATED PAGES
--------------------------------------------------------------------------------
Science and research
USING GENE THERAPY TO REDUCE THE NEED FOR EYE INJECTIONS
In this video, learn about how CERA’s research into gene therapies could
transform the treatment of eye disease
Read More
Research
PREVENTING BLINDNESS AT ITS SOURCE
Researchers at CERA are working to expand gene therapy technology to increase
the number of treatments for blindness-causing genetic disorders.
Read More
* NEW CONSUMER GROUP TO SHAPE FUTURE OF EYE RESEARCH
News
NEW CONSUMER GROUP TO SHAPE FUTURE OF EYE RESEARCH
CERA’S CONSUMER ADVISORY GROUP WILL BRING THE VOICES OF CONSUMERS INTO THE
HEART OF VISION RESEARCH.
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A new Consumer Advisory Group has been appointed to help shape the direction
of research at the Centre for Eye Research Australia and Cerulea Clinical
Trials.
The group will bring the voices of consumers—people living with eye
conditions and their families—into the heart of medical research and
strategic planning.
The Group’s Chair, CERA Board Director Simon Brewin said the new initiative
ensures that consumer insights directly shape research priorities,
development of new treatments for eye disease and the overall direction of
CERA and its fully owned clinical trials subsidiary Cerulea Clinical Trials.
“This is a turning point for CERA. Involving consumers directly in our
research isn’t just the right thing to do—it’s going to make our work more
impactful,’’ he said.
“These are the voices that matter most, and we’re making sure they guide our
research.”
The group includes independent consumers – Jane Cherry, Dr Colleen Lewis, Dr
Ronelle Hutchinson and Daniel Talko –
along with advocates from organisations like Vision Australia, CERA
researchers, and Cerulea Clinical Trials representatives.
The initiative is led by CERA’s Consumer Involvement and Advocacy Lead Kelly
Schulz.
“The strength of this group lies in the diversity of perspectives and lived
experiences each member brings,’’ she said. “This advisory group marks the
starting point for CERA’s broader Consumer Program, laying the foundation for
meaningful, long-term involvement of consumers in our research and strategy.“
The group will provide advice on various aspects of CERA and Cerulea’s work,
from early research design and clinical trial experience to the rollout of
results. This collaboration will help ensure that CERA’s research is both
practical and responsive to the needs of the community.
CERA Managing Director Professor Keith Martin said involving consumers
directly in research was a powerful step forward.
“Their insights will help ensure our work has real-world impact, benefiting
those who need it most,’’ he said. “The launch of the Consumer Advisory Group
marks an important step forward for CERA as it continues to advance eye
research. By embedding consumer perspectives, CERA is strengthening its
commitment to more inclusive, community-driven research—working alongside
other organisations that share this goal.”
* Media contact: Janine Sim-Jones, CERA, jsimjones@cera.org.au +61 420 886
511
Myra McGuinness, Daniel Talko, Kelly Schulz, Luis Alarcon-Martinez, Flora
Hui, Chris Edwards, Ronelle Hutchinson, Jane Cherry, Colleen Lewis, Simon
Brewin and Michelle Gallaher.
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CONSUMER PROGRAM
We work alongside people with lived experience to ensure their insights are
at the heart of advancing eye research.
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Annual Review 2023
BUILDING A CONSUMER CONSCIENCE
Consumer Involvement and Advocacy Lead Kelly Schulz is creating new ways for
CERA’s community to directly influence and improve research at every stage
from the laboratory to the clinic and beyond.
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* PREVENTING BLINDNESS AT ITS SOURCE
Research
PREVENTING BLINDNESS AT ITS SOURCE
RESEARCHERS AT CERA ARE WORKING TO EXPAND GENE THERAPY TECHNOLOGY TO INCREASE
THE NUMBER OF TREATMENTS FOR BLINDNESS-CAUSING GENETIC DISORDERS.
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In recent years, breakthroughs in genetic research have opened the door to
treating inherited retinal diseases (IRDs), such as retinitis pigmentosa,
Usher syndrome and Stargardt’s disease.
Only a few years ago, being diagnosed with one of these IRDs meant a gradual
loss of vision was all but a certainty, but treatments for these diseases are
now on the horizon.
One gene therapy is now available for a rare form of retinitis pigmentosa,
and treatments are being developed for a range of other conditions.
Collectively, IRDs are the most common cause of blindness in working-age
Australians. But despite sharing a name, the genetic mistakes that cause each
IRD are different. A treatment that works for one of the 250 genes known to
cause IRDs will not work for another.
Satheesh Kumar is a graduate researcher in CERA’s Genetic Engineering
Research Unit. He is working with Associate Professor Guei-Sheung (Rick) Liu
to develop new ways of treating disease that would mean more treatments for
different IRDs.
EDITING VS REPLACING
A person’s DNA includes all the instructions that cells need to operate – and
mistakes in this sequence are what cause a person’s body to not operate
properly.
In the case of IRDs, this means vision loss.
“Before starting my PhD, I was an honors student in Queensland and became
interested in gene editing, because there are now a lot of new technologies
that are trying to treat genetic diseases,” Kumar says.
These tools are safe viruses which carry a correct version of the gene, but
they aren’t suitable for every type of disease.
“In traditional gene therapy, a new gene is introduced without fixing the
root cause,” Kumar says.
“The mutated gene is still in the cell, which can sometimes be harmful. And
for a lot of IRDs, the tools we have just aren’t suitable.”
The Genetic Engineering team is using a technology called RNA base editing to
try and correct these mistakes instead.
While DNA is the instruction booklet for cells to function, it is RNA that
carries out these instructions by transmitting their message to build the
different parts of our cells.
“Gene editing that targets RNA enables us to directly correct the mistake
causing the disease,” Kumar says.
“We aim to correct the genetic errors responsible for these diseases at the
molecular level, which could lead to new treatments in the future.”
“WE AIM TO CORRECT THE GENETIC ERRORS RESPONSIBLE FOR THESE DISEASES AT THE
MOLECULAR LEVEL.”
– GRADUATE RESEARCHER SATHEESH KUMAR
TO THE CLINIC
Kumar is hopeful that IRDs once believed to be untreatable will eventually
have therapies, thanks to improved understanding of the genetic causes and
better tools to produce working genes.
“At CERA, when we do research, we are always asking ‘how do we turn this into
a tool for the clinic?’
“When I’m working with Associate Professor Liu, we want to dive deeper into
our understanding of these tools – and alongside many other people around the
world we are constantly learning new things about them.
“Learning more about the basic mechanisms of these tools means we can improve
them, so they can be useful for even more diseases.”
The team’s work requires significant collaborations with others.
“Associate Professor Liu and I work with a lot of collaborators to make sure
these tools are clinically relevant – especially as some of these tools might
also be helpful not just for vison loss but also for hearing loss,” he says.
“I’m excited about the new treatments for many conditions that our research
is working towards.”
Associate Professor Liu’s RNA editing research is supported by an NHMRC Ideas
Grant.
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GENETIC ENGINEERING RESEARCH
CERA scientists are investigating ways advanced gene technology can improve
the treatment of eye diseases that cause blindness.
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* COMMUNITY IMPACT
Stories
COMMUNITY IMPACT
KEITH TEIRNEY, 74, LIVES WITH GLAUCOMA, LEADS AN ACTIVE LIFE AND SUPPORTS
CERA THROUGH COMMUNITY FUNDRAISING.
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“I was diagnosed with glaucoma about 20 years ago,” Keith says.
“Fortunately, I found out early and it doesn’t affect my daily life. I just
have to remember to put my eye drops in every night.”
Glaucoma, the leading cause of irreversible blindness, causes optic nerve
damage.
There is currently no cure, but prescription eye drops, laser therapy and
surgery can help slow or stop vision loss.
CERA’s researchers are investigating how glaucoma can be better treated.
Just two examples include Professor Keith Martin’s research into gene
therapies that aim to protect the optic nerve, and Dr Flora Hui’s research to
determine whether vitamin B3 (nicotinamide) can support glaucoma treatments.
COMMUNITY MINDED
In March, Keith attended CERA’s community forum to learn about the latest
research and treatments being trialled for glaucoma.
“It was also a great chance to talk to others with glaucoma about how they
were coping,” he says.
Afterward, Keith recommended that proceeds from local fundraising activities
at Carnegie Lions Club and Malvern Theatre Company go to CERA.
Professor Martin says CERA is grateful for their support.
“Community fundraising, alongside individual donations, plays a critical role
in enabling us to advance our research.”
Learn more about our community forums and register for our upcoming events.
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GLAUCOMA RESEARCH
Our researchers are among the world’s top scientists investigating the causes
of glaucoma and how it can be better treated. We collaborate across research
areas including gene therapy, mitochondrial research and clinical genetics.
Read More
* MAGGIE SANDLES’ PARALYMPIC DREAM
Stories
MAGGIE SANDLES’ PARALYMPIC DREAM
TEAMWORK IS POWERING MAGGIE SANDLES THROUGH CHALLENGES AND TOWARDS HER DREAMS
OF PARALYMPIC GOLD.
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Maggie Sandles, a 22-year-old para‑triathlete, was hoping to compete in the
2024 Paris Paralympics. At the final qualifier Maggie just missed out, but
she shrugged off the disappointment.
“My next challenge is the World Championships in Spain,” Maggie says.
“Ultimately the goal is to podium at the 2028 Los Angeles Paralympics. That
is my new dream.”
Maggie is as optimistic, determined and driven in sport as she is in life,
and Usher syndrome isn’t holding her back. She sees her condition as a
remarkable opportunity.
Born with Type 1C Usher Syndrome, the most common genetic form of
deaf-blindness, Maggie was profoundly deaf at birth.
Through childhood, she developed balance problems and the gradual onset of
retinitis pigmentosa – a condition associated with Usher syndrome.
Retinitis pigmentosa affects the retina, the light-sensitive layer of tissue
in the back of the eye. As a result, Maggie has decreasing peripheral vision
and trouble seeing any details at night.
While complete blindness is uncommon, Maggie is expecting her sight will
continue to deteriorate.
“The diagnosis was hard,” Maggie says.
“Initially, I could only focus on the fact I was going to be blind by the
time I was 30. Whilst this isn’t true for everyone, that’s what the
ophthalmologist told me.
“My diagnosis was perhaps not delivered gently, but we handled it great as a
family. My mum was very supportive, and my dad dove into the research.”
POSITIVE FOCUS
While Maggie is unfailingly positive, she admits daily life can be a
struggle. Currently, she has around 15-20 degrees of peripheral vision,
compared to a normal 120.
“This means I cannot drive. I am a hyper-independent person, so it’s one of
the hardest things about Usher syndrome,” she says.
In sport, Maggie says she found both an outlet for her energy and a coping
mechanism.
“Sport was a place where I didn’t feel very different. There was always a
bigger challenge, whether it was performance pressure or a hard training
session.”
Maggie says obstacles, like not hearing her coach on the megaphone or a
wobble climbing onto her bike, were soon forgotten.
“Sport taught me resilience and dedication, which are now integral values in
my life.
“While this isn’t how I imagined my life would go, there is a world of
opportunities available that weren’t before. I’m travelling the world and
aiming for the Paralympics and World Championships. I think that’s pretty
cool.”
Bigger dreams: (from left) Lauren and Maggie working together.
BETTER EXPERIENCES
“Maggie sets an incredible example for young people living with inherited
retinal disease,” says Associate Professor Lauren Ayton – a
clinician‑scientist at CERA with a strong research interest in inherited
retinal diseases (IRDs), gene therapy and clinical trials.
“But even she admits that life with Usher syndrome is not what she expected.
“Although there’s currently no treatment for Usher syndrome, breakthroughs
have put many treatments for previously untreatable conditions in reach.”
Gene therapy, stem cell research and regenerative medicine offer avenues for
repairing damaged sensory cells – potentially restoring or improving hearing
and vision.
“CERA’s Genetic Engineering Research Unit is working on a treatment to
prevent vision loss from Usher syndrome,” Associate Professor Ayton says.
“And the work of myself, Dr Tom Edwards and our team is focused on clinical
trials as a tool to improve awareness, support networks and advocacy efforts
for people living with Usher syndrome.
“This is vital to improving access to resources, funding research initiatives
and enhancing the quality of life for individuals and families affected by
Usher syndrome.
“Our ultimate goal is seeing a world where all people with complex conditions
such as Usher syndrome receive best-practice multidisciplinary care.”
BIGGER DREAMS
Maggie says she hopes to be a role model to “inspire others with Usher
syndrome to dream big”.
“I want to go to more than one Paralympic Games and see the world as much as
I can before my sight deteriorates. I hope for driverless cars. I hope for
research breakthroughs that lead to effective treatments and, ultimately, a
cure.
“Inherited retinal diseases affect millions worldwide, causing vision loss
and impacting quality of life.
“The incredible work by CERA researchers using stem cell technology and
gene‑editing techniques offers a beacon of hope for those of us living with
conditions like Usher syndrome.”
Follow Maggie’s journey on Instagram: @maggie_sandles
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* THE END OF GENETIC DISCRIMINATION IN INSURANCE
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THE END OF GENETIC DISCRIMINATION IN INSURANCE
NEW AUSTRALIAN GOVERNMENT POLICY REMOVES BARRIERS TO GENETIC TESTING –
ENCOURAGING EARLY DIAGNOSIS AND PARTICIPATION IN CLINICAL TRIALS THAT COULD
TRANSFORM THE FUTURE OF PERSONALISED MEDICINE AND GENE THERAPY FOR EYE
DISEASES.
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CERA researchers have welcomed the Australian Government’s recent decision to
ban life insurance companies from discriminating against people based on
genetic testing results.
They say the change marks a pivotal shift in healthcare and research and
removes significant barriers that have long discouraged Australians from
undergoing genetic testing.
The change will be a major benefit for people living with conditions like
inherited retinal diseases (IRDs) and age-related macular degeneration (AMD),
where early diagnosis and intervention can be life changing.
The announcement by Assistant Treasurer Stephen Jones, ensures that
Australians can now undertake genetic testing without fear that it will
impact their ability to access life insurance.
The move is expected to encourage greater uptake of genetic testing,
empowering consumers to make informed decisions about their health and
unlocking new opportunities for medical research.
A NEW ERA FOR GENETIC MEDICINE
CERA’s Retinal Gene Therapy Unit co-lead Dr Tom Edwards welcomes the new
policy and says it is a major advancement for genetic medicine and
personalised healthcare in Australia.
“This decision is a major step forward for genetic medicine and personalised
healthcare in Australia.
“For too long, people have been forced to weigh the benefits of genetic
testing against the potential risk of discrimination.
“By removing this barrier, the government is supporting people to make
empowered choices about their health, free from the fear of economic
disadvantage.’’
Genetic testing can provide crucial insights for people living with IRDs that
can lead to early interventions and access to cutting-edge therapies.
Identifying the specific genetic mutations causing these conditions allows
for more targeted treatment approaches, increasing the chances of success and
better outcomes for patients.
“For those with inherited retinal diseases, a genetic diagnosis can open the
door to new therapies that were unimaginable a decade ago,’’ Dr Edwards says.
“It allows us to identify the most effective treatments and tailor them to an
individual’s genetic makeup. Research in this area is moving rapidly, and
knowing your genetic profile can make all the difference.”
However, while this policy change is a significant step forward, Dr Edwards
believes it is just the beginning.
He says the next crucial step is for the Government to provide financial
support for patients to access genetic testing, especially as more gene
therapies become available.
“Without this support, many people may still find it difficult to take that
first step towards a genetic diagnosis and the potential benefits it can
unlock.
“If we want to fully realise the potential of gene therapy, we need to ensure
genetic testing is accessible to all who need it, regardless of their
financial situation.’’
Better access: Dr Tom Edwards believes the announcement is a big step towards
genetic testing for all.
ACCELERATING INNOVATION
The ban on genetic discrimination also has broad implications for medical
research – particularly for gene therapy in clinical trials.
CERA and its ophthalmic clinical trial centre, Cerulea Clinical Trials, are
at the forefront of developing and testing new gene therapies that aim to
prevent or reverse vision loss caused by IRDs.
Cerulea Clinical Trials is bringing cutting-edge research from the lab to
real-world applications and is set to deliver new gene therapy trials
for retinitis pigmentosa and Stargardt’s disease in the next year.
Cerulea CEO Michelle Gallaher says clinical trials are essential to bringing
new treatments to patients, but to participate in a gene therapy trial,
participants typically need to have undergone genetic testing to determine
their eligibility.
By reducing the discrimination and concern associated with genetic testing,
the new policy could lead to increased participation in clinical trials. In
turn, this will provide researchers with more robust data and accelerate the
development of effective therapies for conditions which currently have
limited options.
Gallaher emphasises the importance of this policy change in enhancing
Australia’s position in global ophthalmic research.
“This decision opens the door for more Australians to access genetic testing
without fear of financial repercussions and treatment limitation, and that’s
incredibly important,’’ she says.
“By encouraging more people to take that first step and access genetic
testing, we create a larger, more diverse pool of potential participants for
clinical trials. This, in turn, helps bring more innovative ophthalmic trials
to Australia, ensuring that Australians can access the latest gene therapies
close to home.”
FUTURE FOR PERSONALISED MEDICINE
Gallaher says the changes announced by the Government are a promising sign
that Australia is committed to advancing personalised medicine and ensuring
equitable access to cutting-edge healthcare.
“At CERA and Cerulea Clinical Trials, we are excited about the possibilities
this new policy brings and are dedicated to continuing our work to develop
innovative treatments that improve the lives of those with inherited eye
diseases.
“By ensuring both policy protection and financial support, Australia can
become a leader in making advanced genetic treatments a reality for
everyone.”
Find out more about our gene therapy research.
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Research
FINDING ANSWERS TO UNKNOWN IRDS
A new project is using cutting-edge DNA sequencing technology to find the
genetic cause of not-yet-understood inherited retinal diseases.
Read More
News
CERULEA TO BOOST ACCESS TO SIGHT-SAVING THERAPIES
Cerulea Clinical Trials will bring more international trials to Victoria and
give people living with vision loss and blindness early access to
sight-saving therapies.
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