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October 2022

Join us to talk future gene therapies, treatments for muscular dystrophy, and healing hearts

New gene and cell therapies are on the horizon to treat a range of diseases, including leukaemia and haemophilia, muscular dystrophy, childhood neurological diseases, blindness and deafness, and more.

Increasingly, gene technologies and stem cell science are being used together to understand diseases and deliver new therapies.

Hear about the latest research from experts and ask your own questions at a free public event in Melbourne and online, focused on gene and cell therapies. Read on for details.

This is the second in the Foundation’s new series of public events. In our first event – Future Medicine: Healing the Heart – we heard from three scientists working on treatments for damaged hearts, from the development of tissue patches to injections for treating heart attacks. You can watch a recording of this webinar online. More below.

Melbourne scientist Peter Houweling is growing mini muscles to study muscular dystrophy – a muscle wasting disease that often emerges in childhood. This will assist clinical trials for new treatments. Read on to find out more about this research, which we’re helping to fund, thanks to the generosity of our donors.

Next month we will announce the winners of our 2022 Metcalf Prizes for Stem Cell Research.

And finally, Australian researchers have grown brain cells from stem cells and trained this ‘DishBrain’ to play Pong. This story and more in our regular round up of stem cell news from around the world.

Kind regards,

Dr Graeme L Blackman AO

Chairman, National Stem Cell Foundation of Australia

In this bulletin:

Can we engineer immune cells to treat cancer? Or fix a faulty gene? Melbourne event to explore gene and cell therapies

Bring your questions to a free public event at 5.30pm Tuesday 15 November at the State Library of Victoria.

The convergence of stem cell science and gene therapies are set to impact the future healthcare, with the potential to treat leukaemia, inherited eye diseases, muscular dystrophy, and more.

Gene therapy aims to treat disease by replacing faulty genes or introducing new genes. The field is still in its early days, but there are gene therapies in use, treating some blood cancers and eye conditions.

But what exactly is it? How are stem cells involved? What will it treat? And when? Ask the experts at the Gene therapies and stem cell research: now and in the future public forum.

You’ll hear from a panel of researchers and clinicians sharing their perspectives on treatments for:

  • leukaemia and haemophilia: Dr Ashley Ng, WEHI, Peter MacCallum Cancer Centre and Royal Melbourne Hospital
  • childhood neurological diseases: Dr Sandi Kariyawasam, Sydney Children’s Hospital
  • retinal diseases: Dr Tom Edwards, Centre for Eye Research Australia
  • blindness and deafness: Dr Anai Gonzalez-Cordero, Children’s Medical Research Institute
  • muscular dystrophy: Dr Peter Houweling, Murdoch Children’s Research Institute

This forum will be hosted by Foundation director and stem cell biologist Professor Megan Munsie. You will also hear from advocate and community leader Julie Cini who lost both of her children to the rare genetic condition spinal muscular atrophy.

When: Tuesday 15 November 2022, 5:30PM – 7PM AEDT

Venue: Village Roadshow Theatrette, State Library of Victoria, Swanston Street, Melbourne

Livestream: via our website at  

The event is hosted at the State Library of Victoria by the Australasian Society of Stem Cell Research. The Foundation is supporting the event as part of our mission to provide community education.

Register here.

Turning blood cells into muscle cells to test treatments for childhood muscular dystrophy

Melbourne researcher Dr Peter Houweling is growing mini-muscles in the lab, in order to study muscular dystrophy in children, without the need for invasive and painful tissue biopsies.

“Basically, we take an ordinary blood sample and regress those cells back to their stem cell state. From there, we can encourage the stem cells to develop into skeletal muscle,” explains Peter, who leads the muscle research group at Murdoch Children’s Research Institute.

This project uses recent technological advances that reverse-engineers stem cells out of blood or skin samples from patients with muscle disease. It has been selected for the Foundation’s Matched Funding Program.

Peter’s study is partnering with a clinical trial led by paediatric neurologist Dr Ian Woodcock that is testing the benefit of creatine supplements for children with the rare genetic disorder facioscapulohumeral muscular dystrophy (FHSD).

Like all muscular dystrophies, FSHD is a gradual wasting disease that targets certain muscle groups. Patients may lose the ability to walk, use their arms, talk, or even swallow without aid.

“The clinical trial will assess the effect protein supplements have on young people with FSHD. We’ll use blood samples from the same participants for our stem cell replicas and administer the same supplements in the lab. By comparing results, we can test how accurately we’re modelling real-life muscular dystrophy,” says Peter.

Donations from the public allow the Foundation to keep funding our half of the contribution to important treatment research projects selected for our Matched Funding Program. If you would like to support us with a donation, visit:

Read the full story on our website.

Healthy (left) vs patient-derived skeletal muscle fibres

Healthy (left) vs patient-derived skeletal muscle fibres

Future Medicine: Healing the Heart – watch our webinar

  • Could we boost a failing heart with ‘muscle patches’?
  • Could a stem cell injection help heart tissue regenerate?
  • Can mini hearts in a petri dish solve the mystery of 2,400 babies born with heart disease?
  • And can they explain what COVID is doing to our hearts?

Heart disease kills more than 18,500 Australians a year but that’s going to change. Not tomorrow, but in the coming decades.

Four of Australia’s top stem cell researchers joined a live online audience in August to answer these questions and more in the webinar event Future Medicine: Healing the Heart.

Watch the recording.


Associate Professor James Chong, a researcher at the Westmead Institute of Medical Research and the University of Sydney, and a cardiologist at Westmead Hospital, working on stem cell treatments to repair the damage from heart attacks.

Professor Enzo Porrello, a stem cell scientist at Murdoch Children’s Research Institute working to understand congenital heart diseases and developing tissues to supplement pumping.

Professor James Hudson, a bioengineer at QIMR Berghofer Medical Research Institute finding drug candidates for COVID 19-related heart damage.

Hosted by stem cell biologist and Foundation director and Professor Megan Munsie, from The University of Melbourne and Murdoch Children’s Research Institute.

Stem cell news from around the world

Between newsletters, we share stem cell news on social media:

Here are a few stories we’ve shared recently:

Department of Health and Aged Care: Australia first stem cell research to help adults and children living with joint pain and disfigurement

BBC: Lab-grown brain cells play video game Pong. Paper.

News Medical: Live imaging and cellular barcoding reveal how new stem cells are vetted for quality. Paper

2GB radio: Associate Professor James Chong chats with Dr Ross Walker about the future of stem cell treatments for the heart.

The Conversation: Pregnancy in space: studying stem cells in zero gravity may determine whether it’s safe.

Medical Xpress: Stem cell-gene therapy shows promise in ALS safety trial. Paper

KU Leuven: KU Leuven stem cell biologists create new human cell type for research. Paper

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