Scientists at St Vincent’s Institute of Medical Research have made a significant breakthrough in developing an innovative RNA therapy that could transform the lives of children with rare blood disorders.
The cutting-edge RNA Prime Editing technology has the potential to correct the genetic defect that causes life-threatening conditions such as Fanconi anemia, potentially eliminating the need for risky and costly bone marrow transplants.
Fanconi anemia is a rare, genetic disorder that causes bone marrow failure and affects children from a very young age. Sadly, more than half of the approximately 190 young Australians currently diagnosed with the syndrome will not survive the disease and its related conditions.
The new treatment is progressing through safety studies, in partnership with Maddie Riewoldt’s Vision, an organization dedicated to finding a cure for bone marrow failure syndrome. The goal is to launch a world-first clinical trial for patients.
“Investment in medical research gives St Vincent’s Institute scientists the best chance of maximizing new mRNA technology to develop leading-edge therapy to combat rare childhood diseases like Fanconi anemia,” St Vincent’s Institute of Medical Research Director Professor Thomas Kay said.
This breakthrough research offers real hope for families affected by these devastating diseases.
