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Gene Therapy May Successfully Treat Blood Disorder

β-thalassemia patient blood transfusion-free after lentiviral β-globin gene transfer

THURSDAY, Sept. 16 (HealthDay News) -- Genetic therapy may free patients with severe β-thalassemia from the necessity of receiving regular blood transfusions, according to a letter published in the Sept. 16 issue of Nature.

Marina Cavazzana-Calvo, M.D., of the Assistance Publique-Hôpitaux de Paris, and colleagues report on a patient with severe β-thalassemia, an inherited hemoglobin disorder, 33 months after lentiviral β-globin gene transfer. β-thalassemia patients need regular blood transfusions to boost abnormally low β-globin levels.

The researchers report that the patient, who had required monthly blood transfusions since early childhood, had not needed an infusion for 21 months, and blood hemoglobin had been maintained between 9 and 10 g dl−1, one-third of which contains vector-encoded β-globin. The researchers noted a possible side effect: benign and mild expansion of blood stem cells due to the gene expression-altering effect of the vector used to insert the β-globin DNA.

"The clonal dominance that accompanies therapeutic efficacy may be coincidental and stochastic or result from a hitherto benign cell expansion caused by dysregulation of the HMGA2 gene in stem/progenitor cells," the authors write.

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