Rare Disease Day 2016


With an estimated 350 million people being affected by rare diseases worldwide, 80% of which are caused by faulty genes, the need to fund & support research aimed at increasing our understanding of genetics is the only way we can successfully seek to develop cures & treatment programmes for these rare conditions. Although in recent years we have been able to make significant breakthroughs in our understating of genetic diseases there is still a lag in our ability to develop effective treatments.

Just last week we were thrilled to host the Hon. Pru Goward MP (Minister for Medical Research) who officially opened our latest facility the Vector and Genome Engineering Facility (VGEF).

Under the leadership of Dr Leszek Lisowski (who has joined us from the world renowned Salk Institute in the United States) we look forward to the increased research capability that VGEF will provide to the research teams here at Children’s Medical Research Institute, as well as our paediatric research partners, the Westmead Research Hub, the University of Sydney, and beyond.

In addition to speeding up the gene-related laboratory research that we do on a daily basis, VGEF will boost our efforts to treat children with serious inherited diseases.

The vectors that VGEF develops are microscopic tools for delivering a healthy copy of a gene to the correct tissues and organs inside patients. This technology can speed up research and has the potential to treat and even cure a huge range of genetic diseases.

The immediate benefit of the VGEF is the provision of cutting-edge tools needed by other researchers and clinicians in Australia to progress their work.

To find out more on Vector and Genome Engineering Facility here.

For more info on how you can support the institute click here.

I Support Rare Disease Day



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