A universal gene therapy for Diamond-Blackfan anemia is poised for clinical trials

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.

This article was originally published on MedicalXpress.com

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