I pointed out the new treatment protocol for the sickle cell trait last week. In the interest of complete coverage of hemoglobinopathies, I’ll link to Science’s news article on gene therapy for beta-thalassemia:
?-thalassemia involves a flaw in the gene coding for ?-globin, one of the protein chains that make up the hemoglobin used by red blood cells to transport oxygen. The disease strikes mainly people of Mediterranean and Asian descent. The patient in this trial was typical: Since childhood, he has needed monthly blood transfusions and daily treatments to lower blood iron levels. He was not eligible for a bone marrow transplant, the only cure for the disease, according to Leboulch. In June 2007, when the patient was 19 years old, Leboulch's team removed some of his bone marrow cells and treated them with a modified HIV virus, or lentivirus, carrying a good copy of the ?-globin gene. They infused the repaired cells into the patient; after several months, some of the cells began making ?-globin. The patient's hemoglobin levels are now high enough that for the past 16 months he has not needed blood transfusions and feels well, says Leboulch.
There have been a string of apparent successes for gene therapy lately, including treatment for adrenoleukodystrophy (ALD), and the provision of trichromatic vision in color-blind monkeys.