Researchers have developed a promising new approach for gene therapy of inherited blood disorders that may help overcome therapeutically limiting human stem cell gene transfer efficiency. This method would be applicable to patients with beta-thalassemia, a potentially life-threatening blood disease, as well as other genetic blood disorders, according to a study published in the July 15th issue of Blood. By transplanting beta-thalassemic mice with stem cells treated with MGMT (methylguanine methyltransferase), a drug-resistance gene, researchers were able to subsequently give a chemotherapy drug to the mice, which specifically increased the normal, or globin-expressing cells, to levels that diminished, or in some cases, cured the disease. The transplanted donor stem cells genetically reversed the beta-thalassemia in the mice because the drug-resistant cells assumed production of normal red blood cells in the bone marrow.
"Our finding gives us hope that we might one day be able to help patients with hemoglobin diseases generate healthy blood cells in their own bodies," said Derek Persons, MD, assistant member in the St. Jude Department of Hematology/Oncology, Memphis, TN, and lead author of the paper. "The technique we pioneered will allow us to enrich the population of cells carrying the normal gene by eliminating competing, defective cells, without using radiation or intensive chemotherapy."
Researchers in the study used an oncoretrovirus to transfer MGMT into normal bone marrow cells. The treated cells were then transplanted into beta-thalassemic mice previously given non-myeloablative (non-life-threatening) pre-transplant conditioning with the cytotoxic agents temozolomide (TMZ) and O6-benzylguanine (BG). The transplanted mice were randomly assigned to receive either drug treatment with TMZ/BG (two five-day courses, five weeks apart, beginning seven weeks after transplant) or no treatment.
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