Researchers at St. Vincent’s Institute believe they’re a step closer to curing a rare disorder that triggers bone marrow failure. They’re hoping to start human trials in the next six months using RNA to target and fix a patient’s DNA. Amelia Hawkshaw was 23 when she was diagnosed with Fanconi anaemia, a rare genetic disorder that can trigger tumours, leukemia and bone marrow failure. “There’s an emotional toll and an emotional drain, just from having the disease and worrying about the future”, she says. Bone marrow transplants are an option for the mum of one, but they’re painful and invasive. A treatment researchers believe they can replace with a cure through DNA editing.
A./Prof Andrew Deans said, “It is a lot like science fiction, but actually, we’re trying to make it science reality using a search and replace technology developed at Harvard University; you get to type in the sequence that you want to replace it with”. Researchers at St. Vincent’s Institutes have successfully edited DNA in stem cells.
“Once fixed, those cells could be placed back into a patient to create healthy blood cells and effectively fix the root cause of Fanconi anemia. In many instances, up to 80 or 90% of the cells become corrected, which is way higher than any other types of technology we’ve tried in the past”. Visiting SVI labs, Minister for Economic Growth and Jobs, Danny Pearson said, “This is going to be an absolute game changer, and it proves the strength of our medical research institutes here in Victoria and what can be achieved with partnership”.
Both the Victorian government and Maddie Reiwoldt vision have helped fund the research with local trials are at least two to three years away. However, researchers hope to start human trials overseas in six to 12 months. “Working together with those researchers in the US and another team in Spain, we think we can really bring this technology quite rapidly into use in, in humans, hopefully at at different partner sites, including in Melbourne”, concluded A/Prof Deans.
Story: Nine News Melbourne
Contact: paul.noonan@svi.edu.au
