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The ProblemInherited blood disorders, caused by changes (mutations) in a person’s DNA, are only treatable by bone marrow transplantation. But for many affected children, a suitable donor is not available. For those who receive a transplant, the body's rejection of donor cells can lead to life-threatening complications or failure of the treatment. |
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The ProjectAstrid’s research takes a new approach to tackling inherited blood disorders: ‘editing’ the genetic code to repair ‘faulty’ DNA sequences, so as to restore normal function of a patient’s own bone marrow cells (meaning a donor is no longer needed). “I have developed several new tools for DNA editing that this project will test in mice, so we can verify the tools’ effectiveness in a living animal. Using DNA editing to change the colour of a fluorescent marker, we will work to determine the best strategies for a safe and effective gene therapy for Australian patients,” says Astrid. |
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Dr Astrid GlaserDr Astrid Glaser is a gene editing expert driven to cure rare and debilitating diseases. Before joining SVI’s Genome Stability Laboratory in 2018, Astrid began her scientific training at the University of Vienna and completed her PhD at Murdoch Children’s Research Institute in Melbourne. Astrid’s expertise has been central to establishing gene editing technology at SVI, which is now used in many research projects. Learn more about Astrid's research in this short video. |