Efforts to develop a gene therapy for Diamond-Blackfan anemia (DBA) - a rare, life-threatening disorder in which bone marrow cannot make mature, functioning red blood cells - have been hampered by the fact that at least 30 different genetic mutations can cause the disorder.
- By NANCY FLIESLER | Boston Children's
A team led by researchers at Harvard Medical School has now cleared that obstacle by developing a universal gene therapy for DBA, one designed to correct the bone marrow defect no matter the patient's specific mutation.
The experimental therapy is ready to test in clinical trials, the team reports Nov. 11 in Cell Stem Cell.
"This is one of first examples where we can develop a gene therapy that can target dozens of mutations with a single vector," said senior author Vijay Sankaran, the HMS Jan Ellen Paradise, MD Professor of Pediatrics at Boston Children's.
