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Joan Stephenson, PhD

JAMA. 2002;287:180.

	Since this article does not have an abstract, we have provided the first 150 words of the full text and any section headings.

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).

A new gene therapy technique for sickle cell disease succeeded in correcting the defect in mice. Sickled cells remain in control mice that received a sham treatment; red blood cells are normal in mice receiving a gene to treat the defect. (Photo credit: Copyright 2001, Science; http://www.sciencemag.org)

In SCD, abnormal -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 -globin, the investigators designed a -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 . . . [Full Text of this Article]