Posted 18 July 2024
SVI and Maddie Riewoldt’s Vision (MRV) proudly hosted Deputy Premier Ben Carroll, Minister for Medical Research, and Sheena Watt MP for a tour of the Genome Stability Lab, headed by Associate Professor Andrew Deans.
Until recently, gene editing technologies were science fiction but now, Andrew’s team is developing cutting edge gene edition technologies that hope to prevent children and young adults from dying of inherited bone marrow failure syndromes.
Their focus is to locate and edit disease-causing mutations in bone marrow stem cells to ensure a lifelong source of normal blood cell production. This game changing research, supported by MRV, will improve stem cell transplant success rates by removing the need for an unrelated donor and the toxic conditioning that is currently needed for a successful bone marrow transplant.
About bone marrow failure syndromes
Bone Marrow Failure Syndromes are a collection of medical conditions where the bone marrow stops working or works insufficiently.
This means that bone marrow stem cells can’t produce enough healthy red blood cells, white blood cells and platelets to meet the body’s daily requirements.
Bone Marrow Failure Syndromes may be acquired, meaning a previously healthy person can develop bone marrow failure. Bone Marrow Failure Syndromes can also be inherited in families where there may be a history of the condition.
Inherited bone marrow failure can lead to additional health challenges, including heart, skeletal malformations, kidney, eye and ear issues, and an increased risk of both solid organ and blood cancer.