Children's Medical Research Institute founded

The idea of a Children’s Medical Research Foundation was formulated by Sir Lorimer Dods and Dr. John Fulton. The Foundation (later renamed Institute) was established in 1958 with funds raised by Australia’s first telethon and a large public campaign.

Pioneered microsurgery

We pioneered microsurgery techniques to help repair tiny blood vessels and organs in infants and children, with the techniques developed here now widely used around the world. We also developed a better understanding of donor organ rejection in children and consequently improved the survival of infants following organ transplantation.

Jeans for Genes

We helped lead the genetic revolution by establishing The Gene Therapy Research Unit, a joint venture with The Children’s Hospital at Westmead. In collaboration with the Children's Hospital in Paris, the Gene Therapy Research Unit conducted the first-ever gene therapy clinical trial for a genetic disease in Australia.

To fund this new field of research and educate the public about genes and genetics, we created the Jeans for Genes campaign, which was later replicated by other charities in the US and UK.

ProCan Launches Moonshot

The ACRF International Centre for the Proteome of Human Cancer (ProCan) was established at CMRI, quickly attracting the interest of the US Cancer Moonshot program, becoming its first international member. ProCan's 7-year plan is to analyse tens of thousands of examples of all types of cancer from all over the world to develop a library of information to advance scientific discovery and enhance clinical treatment worldwide.

Blueprint of Embryo Development Completed

Decades of work by Professor Patrick Tam and his international collaborators led to this significant publication in Nature. As Prof Tam explains,  “What we have learned from studying the genome during development is that each gene—each piece of music—does not act in isolation. It is more a symphony. Many genes work together to create an orchestra of signals to coordinate the growth and development of an embryo. We have now gone beyond studying individual genes to studying the entire gene network and how errors in the network can lead to disease. This knowledge also helps make stem cell therapy possible. A stem cell can become any type of cell. But, like a kindergartner let loose on the playground, you have no idea where she will end up. By understanding the signalling networks, we can direct stem cells into useful paths, to become liver cells or retinal cells. We can generate desired cell types from stem cells for regenerative therapy, to restore missing or malfunctioning cells."

First gene therapy for genetic blindness

Professor Robyn Jamieson led the delivery of Australia's first gene therapy for a genetic eye disease, which was also Australia's first TGA approved gene therapy treatment. The therapy was delivered as part of Ocular Gene and Cell Therapies Australia (OGCTA), a new collaboration involving the Genetic Eye Clinic at Sydney Children’s Hospitals Network (SCHN), the Eye Genetics Research Unit and Stem Cell Medicine Group at the Children’s Medical Research Institute (CMRI), and the Save Sight Institute at Sydney Eye Hospital and University of Sydney.