GENE THERAPY: Gene Gemisch Cures Sickle Cell in Mice

doi:10.1126/science.294.5550.2268a

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Science 14 December 2001:
Vol. 294. no. 5550, p. 2268
DOI: 10.1126/science.294.5550.2268a

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GENE THERAPY:
Gene Gemisch Cures Sickle Cell in Mice

Eliot Marshall

On page 2368, a team reports having used an HIV-based vector to cure sickle cell disease in mice. Researchers think this is one of several signs that gene therapy could become a reality for this disease, although there is still a lot of work to be done.

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REPORTS Correction of Sickle Cell Disease in Transgenic Mouse Models by Gene Therapy: Robert Pawliuk, Karen A. Westerman, Mary E. Fabry, Emmanuel Payen, Robert Tighe, Eric E. Bouhassira, Seetharama A. Acharya, James Ellis, Irving M. London, Connie J. Eaves, R. Keith Humphries, Yves Beuzard, Ronald L. Nagel, and Philippe Leboulch (14 December 2001)
Science 294 (5550), 2368. [DOI: 10.1126/science.1065806]
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