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Scientists in the United States, France, and Canada have used a new gene therapy technique to correct sickle cell disease (SCD) in mice (Science. 2001;294:2368-2371).
In SCD, abnormal β-globin molecules adhere to each other and form long fibers that deform red blood cells into the sickle shape that is the disease's hallmark. To correct the defective gene that encodes the abnormal β-globin, the investigators designed a β-globin gene that encodes a sickling-resistant protein and introduced it into modified retrovirus. Other researchers had earlier demonstrated that the latter—a lentivirus to which a piece of HIV-1 had been added—is an efficient vehicle for carrying a gene into resting stem cells, such as the hematopoietic stem cells in bone marrow.
Stephenson J. Sickle Cell Gene Therapy. JAMA. 2002;287(2):180. doi:10.1001/jama.287.2.180