Fanconi Anaemia is the most common cause of inherited bone marrow failure with a median onset of only eight years of age. Bone marrow failure is fatal in Fanconi Anaemia except for one life saving therapy: a bone marrow transplant. However, transplants have risks to children, including life threatening toxicities and increased risk of developing secondary cancers. Such risks would be substantially reduced by using gene therapy, which corrects the genetic mutation that causes the bone marrow failure. Recent published clinical trials have proven gene therapy as a potential curative treatment for bone marrow failure in these patients. This project will investigate a new gene editing technology (Prime Editing), with the aim of efficiently correcting Fanconi mutations. If successful, this would provide a new alternative therapeutic option for preventing bone marrow failure in this patient group.
2018-2023 (Grant-in-Aid): Using induced pluripotent stem cells to find causes and cures for bone marrow failure in children and young adults. Professor Andrew Elefanty, Murdoch Children’s Research Institute. Bone Marrow Failure Syndromes may be ...
Read more2018-2021 (Grant-in-Aid): Identifying therapeutics which can be repurposed for the treatment of Myelodysplastic Syndromes and other bone marrow failure disorders. Associate Professor Amee George, The Australian National University. There are ...
Read more2018-2020 (Grant in aid): Discovering new genes and mutations that cause failure of bone marrow neutrophil production. Professor Graham Lieschke, Australian Regenerative Medicine Institute/Monash University and The Royal Melbourne Hospital. ...
Read more2019-2021 (Grant-in-Aid): Establishing an in vivo humanised mouse model for telomere related Bone Marrow Failure Syndromes. Professor Tracy Bryan, Children’s Medical Research Institute. Human genetic information is packaged into discrete bundles ...
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