A preclinical trial of next generation gene editing for the prevention of bone marrow failure in Fanconi Anaemia

2023- 2025 (Fellowship): The inaugural Captain Courageous Fellowship. A preclinical trial of next generation gene editing for the prevention of bone marrow failure in Fanconi Anaemia. Dr Astrid Glaser at the Genome Stability Unit of St Vincent’s Institute for Medical Research.

This research aims to provide an alternative treatment to Fanconi Anaemia, a serious genetic disorder that leads to bone marrow failure. Currently the only treatment for Fanconi Anaemia is a bone marrow transplant from a donor, with high risk of complications.

Dr Astrid Glaser is a gene therapy expert. Her goal is to develop ways to ‘edit’ errors in a person’s DNA to ultimately cure inherited diseases. Astrid’s current focus is gene editing techniques with the goal of repairing the underlying changes in a person’s DNA that cause inherited Bone Marrow Failure Syndromes.

Inherited conditions are particularly difficult to treat because they are caused by changes (mutations) in the person’s individual genetic blueprint – their DNA. Gene editing technology offers the potential for scientists to locate and fix the mutations that drive disease in this DNA blueprint – a goal which would ultimately cure many genetic disorders.

Dr Glaser’s Fellowship will enable the development of new gene editing tools to repair the DNA mutations that cause Fanconi Anaemia, the most common inherited Bone Marrow Failure Syndrome.

“Gene therapy holds the potential to rescue bone marrow failure without the dangerous complications associated with a transplant from a donor,” Dr Glaser explains. “My hope for this research is that we will be able to expand our work to other inherited Bone Marrow Failure Syndromes, laying the foundation for a clinical trial to improve the lives of Australian patients and their families.”

This exciting research is possible thanks to our partners at Captain Courageous. “We are very excited to award Dr Glaser’s research with the inaugural Captain Courageous fellowship. This research is a very important key to understanding Bone Marrow Failure Syndromes. We can’t wait to hear the outcome.”