AIDS virus to treat blood disorder, beta thalassemia
The 18-year-old suffered from the inherited condition beta thalassemia and required monthly blood transfusions from the age of three. He felt ill and drained of energy because his system could not produce red blood cells due to a faulty haemoglobin gene, reports the Daily Mail.
The only known cure was stem-cell transplantation, but few patients are able to find a suitable donor. Now scientists have found a new potential cure using gene therapy, according to the journal Nature.
The team, led by Marina Cavazzana-Calvo of the University Paris-Descartes, extracted haematopoietic stem cells (HSCs) from the young patient’s bone marrow. These are the cells that go on to produce all types of blood cell. The cells were mixed in a test tube with an engineered version of the HIV virus that carried the correct version of the haemoglobin gene.
The patient had chemotherapy to destroy his remaining stem cells and then the treated cells were injected into his system. Within a year, the teenager was able to stop receiving blood transfusions. Although he is mildly anaemic he has remained stable for 22 months since then.
Study co-author Philippe Leboulch of the Harvard Medical School said the treatment had been “life-changing”. He added: “Before this treatment, the patient had to be transfused every month. Now he has a full-time job as a cook.” (Agencies)