A team led by Harvard Medical School researchers has developed a universal gene therapy for Diamond-Blackfan anemia (DBA) that shows promising efficacy regardless of a patient's specific mutation.
Diamond-Blackfan anemia (DBA) is a life-threatening condition in which bone marrow does not produce functional mature red blood cells. Additionally, gene therapy treatment methods have previously been unsuccessful because at least 30 different genetic mutations cause the disorder.
The gene therapy developed by the researchers, which is published in Cell Stem Cell, is now ready to test in clinical trials.
"This is one of the first examples where we can develop a gene therapy that can target dozens of mutations with a single vector," said Vijay Sankaran, Professor of Pediatrics at Boston Children's.
During lab testing, the therapy showed a substantial increase in red blood cell production over other current treatment methods. In addition to treating DBA, the research could have implications for the development of other novel gene therapies. "This could open up avenues for a whole other set of blood diseases," Sankaran added.