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 Blood, 1 November 2004, Vol. 104, No. 9, pp. 2697-2703.
Prepublished online as a Blood First Edition Paper on July 15, 2004; DOI 10.1182/blood-2003-10-3717.

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GENE THERAPY

Targeted retroviral transduction of c-kit+ hematopoietic cells using novel ligand display technology

Anil Chandrashekran, Myrtle Y. Gordon, and Colin Casimir

From the Department of Haematology, Faculty of Medicine, Imperial College London, Hammersmith Hospital Campus, London, United Kingdom.

Gene therapy for a wide variety of disorders would be greatly enhanced by the development of vectors that could be targeted for gene delivery to specific populations of cells. We describe here high-efficiency targeted transduction based on a novel targeting strategy that exploits the ability of retroviruses to incorporate host cell proteins into the surface of the viral particle as they bud through the plasma membrane. Ecotropic retroviral particles produced in cells engineered to express the membrane-bound form of stem cell factor (mbSCF) transduce both human cell lines and primary cells with high efficiency in a strictly c-kit (SCF receptor)-dependent fashion. The availability of efficient targeted vectors provides a platform for the development of a new generation of therapies using in vivo gene delivery. (Blood. 2004;104: 2697-2703)


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