Projects
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Precision gene editing for the treatment of Fanconi Anaemia. 

2021-2024 (Grant-in-Aid): Precision gene editing for the treatment of Fanconi Anaemia. Dr Lorna McLeman, St Vincent’s Institute of Medical Research.

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.

Related Projects

Establishing an in vivo humanised mouse model for telomere related bone marrow failure syndromes.

2019-2021 (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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Identifying therapeutics which can be repurposed for the treatment of Myelodysplastic Syndromes and other bone marrow failure disorders.

2018-2021 (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 ...

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Towards targeted treatments for Fanconi Anaemia 

2017 – 2019 Fellowship / 2017 Grant-in-Aid, Towards targeted treatments for Fanconi Anaemia, Associate Professor Wayne Crismani, St Vincents Institute for Medical Research Fanconi Anaemia is an inherited disorder which can lead to bone ...

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Outcomes of Haematopoietic Stem Cell Transplantation for Paediatric Patients with Severe Aplastic Anaemia (SAA) and Bone Marrow Failure (BMF) Syndromes.

2021-2022 (Grant-in-Aid): Outcomes of Haematopoietic Stem Cell Transplantation for Paediatric Patients with Severe Aplastic Anaemia (SAA) and Bone Marrow Failure (BMF) Syndromes. Dr Steven Keogh, Australasian Bone Marrow Transplant Recipient ...

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