Thanks to some generous donors, donations made to Jeans for Genes Day tomorrow, Friday 7 August, will be doubled – and help researchers at the Children’s Medical Research Institute (CMRI) continue their important work in gene therapy.
Jeans for Genes raises funds for the work being done at CMRI where scientists are fighting to keep their labs open during the pandemic in order to make history in the field of gene therapy.
There are thousands of genetic diseases that have no treatment or cure. Gene therapy hopes to change that by using one injection to correct the fault in a gene and permanently cure a child.
CMRI has one of the world’s leading gene therapy units led by Professor Ian Alexander. Paediatrician Dr Kate Mullany works with children who need transplants and recently joined the gene therapy team after becoming frustrated that she couldn’t give more hope to parents.
“As a doctor you are meeting these families in an emergency, on the worst days of their lives,’’ Dr Mullany said.
“They have so much hope that a transplant will give their child a new chance at life. Whilst a transplant is life saving and life changing in many ways, it does not fix the underlying genetic defect and these kids still have lots of health challenges. A liver transplant is a really big band-aid, it can help, but it’s definitely not a cure.
“Why I came into research is that so often I’m working with a family, hand-in-hand, and I know that I can’t offer them a treatment or a cure. They go through so much, they are so brave and they still keep smiling and I couldn’t help but feel there had to be a better way to help these children and their families. When I heard about the gene therapy research team at the CMRI I thought, ‘I have to be a part of this, I want to be part of history in the making’. I’m really excited for the potential. The belief that we will one day be offering cures is what keeps us all ticking.’’
One of her patients was Charlize Gravina, who had a successful transplant and gave her liver to CMRI for research. Charlize’s mum, Julie, knows that it is indeed a band-aid and wants a better future for her daughter and others, after losing her son Isaac to the same genetic liver disease, called Propionic Acidemia.
“The liver transplant has saved Charlize’s life, but we live in fear of rejection, and she still has a limited life span,” Julie said. “If she could have been given gene therapy, her life span would be normal.
“This pandemic has given people a taste of what we live with, waiting for a cure. Maybe people will now understand the importance of research.’’
For the first time in the 27-year history of Jeans for Genes, there won’t be volunteers on the street in Australia’s capital cities. Everyone is being encouraged to stay home, stay safe and donate online.
All donations made on Friday will be doubled by some generous donors; to donate, head to jeansforgnees.org.au
CMRI pioneered microsurgery, immunisations against lethal childhood illnesses, and care for premature babies, all of which has improved the lives of countless Australian children over the last 61 years. Today, CMRI is an independent institute and the site of world-leading research in the areas of cancer, neurobiology, embryology and gene therapy. CMRI is affiliated with the University of Sydney and is a founding partner of Luminesce Alliance and the Westmead Research Hub. Find out more at www.cmri.org.au. This not-for-profit institute is funded by competitive grants and a community of supporters who participate in events like Jeans for Genes Day, which is held on the first Friday in August. Visit www.jeansforgenes.org.au.