Steps toward generating new molecular therapies for Bone Marrow Failure Syndromes

2020 – 2024 (Alex Gadomski Scholarship): Steps toward generating new molecular therapies for Bone Marrow Failure Syndromes, Ariel Simpson, Menzies Institute for Medical Research and University of Tasmania.

The only established treatment for Bone Marrow Failure Syndromes is a bone marrow transplant, which has a high risk of complications. Patients with inherited bone marrow failure also commonly experience symptoms outside the bone marrow that cannot be improved by a transplant.

This project takes multiple steps towards developing new therapies for Bone Marrow Failure Syndromes to improve the safety of transplants and address complications that occur outside the bone marrow: 

1) achieve correction of changes (mutations) in the DNA that cause bone marrow failure using new ‘gene editing’ technology,

2) uncover how blood cells develop from bone marrow, and

3) develop a strategy for the generation of a ‘universal donor cell line’ – a product that could be used safely for transplantation instead of donor bone marrow.

This work may give rise to new therapies which are sorely needed to improve the lives of patients and their families.