Diamond-Blackfan anemia (DBA) is a potentially life-threatening condition that can cause severe anemia and other abnormalities. It’s a rare genetic blood disorder that occurs when the bone marrow fails to make red blood cells.
Currently, the only cure is a stem cell transplant, but this is only a viable option for select patients. Otherwise, children with DBA require lifelong follow-up care to manage symptoms, such as steroids and blood transfusions.
A new study published in the journal Cell Stem Cell provides strong support for the first-in-human universal gene therapy trial for DBA through regulated GATA1 expression. Notably, it’s one of the first examples of treating 30 different genetic mutations with a single vector. This clinical-grade vector is ready, and the team hopes to take it to clinical trial next.
More information:
Richard A. Voit et al, Regulated GATA1 expression as a universal gene therapy for Diamond-Blackfan anemia, Cell Stem Cell (2024). DOI: 10.1016/j.stem.2024.10.012
Citation:
New research supports the first in-human universal gene therapy for Diamond-Blackfan anemia (2024, November 11)
retrieved 11 November 2024
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